hemophilia-newyork

Group Meeting

Come connect with men & women who

have bleeding disorders, and women who are carriers!

(Ages 18 & older)

Wednesday, May 23rd

There will be dinner

&

a Guest Speaker

Topic:

New Horizons for Aging Adults

Location: Public House Restaurant

Handicap Accessible

You need to RSVP by May 21st with Annie

annhany@bestweb.net

212-682-5510

2012 BDAN Fall Family Retreat

October 6 - 8, 2012 (Columbus Day weekend)

We know living with bleeding disorders is a challenge. That's why we created the Fall Family Retreat! Open to families and adults with bleeding disorders, the Retreat is an informativeand fun 3 day retreat in New York's scenic Adirondack Mountains.

The Family Retreat is FREE and includes:

- Separate adult and youth activities.

- Hands on information programs.

- The chance to make new friends who share your challenges!

Camp Aldersgate features comfortable heated and furnished cabinson over 100 acres of land bordering two lakes.

Space is limited, so apply early! A $50 deposit is asked to reserveyour place and will be returned to you when you arrive. Gas money is provided if you need. The deposit can be waived for those who qualify.

Bob Graham 315 - 396 - 2944

Email: bobgraham04@msn.com

Learn more or Apply at www.bdaninc.org

Thanks to Everyone Who
Help Support Our Fight
Against Hemophilia at the

May 6, 2012 Day at the Races

A special thanks to Kathy Honreich,

who organized the event.

World Hemophilia Day

April 17

Close the Gap

An estimated 1 in 1000 women and men has a bleeding disorder. However, 75% still receive very inadequate treatment or no treatment at all. What will it take to close this gap? What will it take to close the gap?

On World Hemophilia Day 2012 help us spread the message to “Close the Gap” of care around the world. Together, we can work towards a day when treatment will be available for all globally.

The reality is that most people with hemophilia or other bleeding disorders do not receive adequate diagnosis, treatment, and management for their conditions. This is important whether good treatment is already established but needs to be protected or where treatment needs to be improved.

The WFH would like to hear how you and your organization or treatment centre marked World Hemophilia Day. Please send an update, along with a few high resolution photos to sford@wfh.org by May 18.

Join the international bleeding disorders community on April 17 to mark World Hemophilia Day. Together, we will Close the Gap.

Thanks to NY State Bleeding Disorders Advocacy Coalition NY does not have high cost copayments or deductibles for pharmacy tiers for high cost drugs such as factor.

Read this important article:
www.nytimes.com/2012/04/13/health/states-seek-to-curb-exorbitant-drug-costs-incurred-by-patients.html?pagewanted=all

Inspiration Biopharmaceuticals Announces Filing of Biologics License Application (BLA) for IB1001, a Recombinant Factor IX Product for People with Hemophilia B

Approval of IB1001 would offer people with hemophilia B

a choice of recombinant therapies

Cambridge, Mass., April 17, 2012 – Inspiration Biopharmaceuticals, Inc. today announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the approval of IB1001, an intravenous recombinant factor IX (rFIX) protein for the treatment and prevention of bleeding in individuals with hemophilia B.

Based on the terms of its agreement with Ipsen (Euronext: IPN; ADR: IPSEY), Inspiration will receive a $35 million milestone payment associated with its filing of the BLA. In return, Inspiration will issue a convertible note to Ipsen, bringing Ipsen's fully diluted equity ownership position in Inspiration to approximately 43.5%.

Approval of IB1001 would represent a significant advance for the hemophilia community, including the estimated 75% of people living with hemophilia worldwide who do not have adequate access to currently available treatments. IB1001 would be the first recombinant therapy for people with hemophilia B introduced in more than 15 years.

Regulatory review is now pending in both the

U.S.

and Europe. Inspiration's Marketing Authorization Application for IB1001 was accepted by the European Medicines Agency in September 2011. The company is currently finalizing plans for additional regulatory filings and preparing for the commercial launch of IB1001.

"The introduction of recombinant factor IX was an important step forward in the treatment of hemophilia B, but a single product is not sufficient to meet the needs of all people affected by this disease," said John P. Butler, Inspiration's Chief Executive Officer. "At Inspiration, we are solely focused on the needs of people with hemophilia, and we are proud of the progress we have made towards bringing forward a new choice of treatment for patients. We plan to work closely with regulatory authorities both here in the

U.S.

and around the world to make IB1001 available as broadly and rapidly as possible."

The IB1001 BLA filing includes a comprehensive set of pharmacokinetics safety, and efficacy data from a Phase 3 clinical trial in people affected by hemophilia B. A surgery substudy was also included.

John Taylor, Co-Founder and Chairman of Inspiration, stated, "With sons affected by hemophilia B, Inspiration co-founder Scott Martin and I are acutely aware of the impact of limited treatment options on families. We believe more choice can help us achieve our mission to broaden access to care—increasing the supply of products, driving healthy competition, and supporting prophylaxis, which is rapidly becoming the standard of care in hemophilia. We believe that the approval of IB1001 will be the first step in our journey to make broader access to care a reality for thousands of individuals with hemophilia."

About IB1001
IB1001 is an intravenous rFIX product being developed for the treatment and prevention of bleeding in individuals with hemophilia B. IB1001 has completed pivotal Phase 3 clinical studies that support the filling of the BLA, conducted at study sites in the

U.S.

, Europe and

India

.

To date, IB1001 has been well tolerated by patients and pharmacokinetic (PK) analyses have demonstrated non-inferiority to the one approved rFIX product currently marketed for the treatment of hemophilia B. Confirmatory PK studies reported at the 53rd Annual Meeting of the American Society of Hematology showed no evidence of development of inhibitors during treatment periods ranging from 4 to 18 months.

Results recently presented at the 5th Annual Congress of the European Association for Haemophilia and Allied Disorders demonstrated that IB1001 provided effective surgical hemostasis in study participants with hemophilia B undergoing major surgical procedures.

About Inspiration Biopharmaceuticals
Inspiration Biopharmaceuticals is exclusively dedicated to developing treatments for hemophilia, with a primary mission to broaden access to care by providing safe and effective recombinant therapies and advancing innovation for people living with hemophilia. Inspiration has a broad portfolio of recombinant hemophilia product candidates, which includes one under review by the FDA and EMA for marketing approval in the

U.S.

and Europe, one in late-stage clinical development, and two preclinical programs.

Inspiration's lead product candidates are IB1001 and OBI-1, an intravenous recombinant porcine factor VIII (FVIII) product being developed for the treatment of individuals with congenital hemophilia A who have developed inhibitors against human FVIII, and for individuals with acquired hemophilia. Inspiration has earlier-stage development programs focused on human recombinant factor VIIa (rFVIIa) for individuals with either hemophilia A or hemophilia B who have developed inhibitors or those with factor VII deficiency, and human recombinant FVIII for individuals with hemophilia A.

Inspiration's senior management team has broad experience and expertise in hemophilia product development, biologics manufacturing and the successful commercialization of products to treat hemophilia and other rare diseases.

In January 2010, Inspiration entered into a strategic agreement with Ipsen (EURONEXT: IPN; ADR: IPSEY), leveraging the combined expertise and resources of the two companies, to develop a broad portfolio of hemophilia products. As announced in late August 2011, Ipsen and Inspiration extended their agreement to create a hemophilia business unit structure that will act as the exclusive sales organization for all hemophilia products commercialized under the Inspiration brand in Europe. For further information on Inspiration, please visit www.inspirationbio.com.

April is Donate Life Month

Source of life: Advocates push for organ donor awareness

Established in 2003, the designated month raises awareness about the importance of lives saved through the donation of organs, as well as tissue, marrow and blood.

Government data shows about 112,000 people in the United States are waiting for an organ transplant — more than 1,800 in Wisconsin alone. Every 11 minutes another person’s name is added to the list.

As of October 2011, there were 100 million designated donors in the U.S., just more than 40 percent of the population age 18 and above, according to donatelife.net. And, according to the University of Wisconsin Organ Procurement Organization in Madison, more than half of Wisconsinites already are listed as donors.

One organ donor can save the lives of eight people.

How to donate

There are more ways to donate organs than designating the decision on the back of your driver’s license, said Jill Ellefson, executive director for the last three ears of the UW Organ Procurement Organization in Madison.

“We also have an online registry yesIwillwisconsin.com, which is an online version of signing your driver’s license,” she said. “And what’s great about that is we can access that database 24/7. The problem with a driver’s license is that sometimes that’s not available at the hospital and it’s sometimes a challenge to find that document.”

Since the yesIwillwisconsin.com registry opened in March 2010, nearly 30,000 people have registered as donors. People, ages 15 ½ years or older, with an orange donor dot on their license or state ID should still sign up to ensure their wishes will be honored.

Potential donors also can document their wishes on an advanced directive, which again is not always readily available.

Another option is living donations where a donor gives one of their two kidneys or a segment of their liver. Over half the 112,000 on the waiting list in the country are waiting for a kidney.

In addition to signing onto a registry, Ellefson said, it’s important to share that decision of donation with the family. “Even if you sign your license or signed up on the registry, your family still likes to know and it’s better for them to hear your decision from you instead from us.”

Being on the donor registry is a legal decision that cannot be changed by anyone else with the exception of those under 18.

To read the rest of this article please go to:

www.postcrescent.com/apps/pbcs.dll/article?AID=/201204090422/APC04/120406075

HANY Scholarship

HANY scholarship applications are available. If you need one and you meet the requirements, you can give us a call at 212-682-5510.

These are some of the requirements to qualify:

You must be registered with HANY.

You have to be a full-time student at an accredited college, university, graduate school, or certified trade or vocational school for the coming school year.

Scholarships are available to persons with inherited genetic bleeding disorders (as diagnosed by a board-certified hematologist) and their children. Considerations will also be given to females who are carriers attested to by a board-certified hematologist.

You must live within the fourteen counties of southeastern New York (the five boroughs of New York City, Long Island, Dutchess, Orange, Putnam, Rockland, Sullivan, Ulster and Westchester counties) served by HANY.

This video was geared for a fundraising campaign that has ended.

For updates, you can check out the

Facebook page: www .facebook.com/STBhemo

There will be a more comprehensive all-purpose

video available next month.

This video is not intended to deliver any medical advice.

You should consult your physician or local treatment center

before pursuing any course of treatment.

Action Alert: Support Gov. Cuomo's plan to issue an Executive Order to establish a NY Health Insurance Exchange!

Due to inaction of the part of the State Senate, establishing a New York Health Insurance Exchange through the final State Budget is no longer possible.

However, media reports now indicate that Governor Cuomo does not plan to turn his back on the millions of small businesses and families in New York who are being crushed by the cost of health insurance. Instead, he plans to do what's responsible and issue an Executive Order to establish a New York Health Insurance Exchange!

Call Governor Cuomo's office TODAY to thank him

and support his plan to issue an Executive Order

to establish a New York Health Insurance Exchange this week!

CALL (518) 474-8390

No matter what happens in the Supreme Court, New York still needs to fix its insurance market. While an Executive Order cannot accomplish everything that legislation would have done, it will still move the state government forward in helping New Yorkers in need.

An Exchange is vital for New York for three reasons:

With an Exchange, New York's small business health insurance costs will drop by 22%. If small group premiums drop this significantly, businesses will be able to hire new workers, instead of struggling to keep up with skyrocketing health premiums. In addition, folks who buy insurance on their own on the individual market will see costs drop by 66%! (See Urban Institute study on the impact of an Exchange in NY). Lower health costs will make New York's economy stronger.

There are currently over 2.8 million New Yorkers living without health insurance because they cannot afford it. An Exchange will make quality, affordable health insurance options available to all, and 1 million New Yorkers are estimated to newly gain coverage as a result.

Establishment of an Exchange will allow New York State to draw down millions in federal funds to help get our Exchange up and running and helping New Yorkers as soon as possible.

Don't wait! Call today!

The New York City Hemophilia Chapter invites you to join us for a day of food, friends and fun at our Spring Event at the beautiful Bronx Zoo on Sunday, April 22, 2012 at 12 noon. The event ticket includes admission to the zoo, a BBQ buffet lunch with hamburgers, hot dogs and other treats, a wide range of rides and attractions, and more. This will also be one of the first times the zoo's new baby giraffe can be viewed by the public!

There is no charge for this event - it's free!. However, participation is limited to people with bleeding disorders and members of their immediate family.

Please click here to reserve your tickets for the Spring Event at the Bronx Zoo.

If you have questions or need more information, please contact Jeri Krassner at jeri.krassner@nyhemophilia.org or at (917) 597-7256.

We've Moved

We have moved to a temporary office until July.

Our main number 212-682-5510 remains the same or

you can call us at 646-237-6956.

Our temporary fax number is: 646-253-1258,

please call us to let us know when you will be faxing a document.

SUMMER CAMP SESSIONS

We have posted 2012 camp sessions at any
of the three specialty camps nearest to our area.
All three camps provide on-site medical care and supervision.

For more information go to:

www.hemophilia-newyork.org/campinformation.html

                 Hole in the Wall          Double H Ranch          Camp High Hopes
                       Ashford, CT                  Lake Luzerne, NY              Brantingham, NY

Albany Days
March 18-19, 2012

URGENT: BIOLOGIC RECALL INFORMATION

Initiated by: Baxter Healthcare
Event Id: 151
Event Date: 03-14-2012

Therapy

Gammagard Liquid [Immune Globulin Intravenous (Human)] 10%

NDC Number: 0944-2700-06

Lot Number: LE12LG70AC

Size: 20g

Packaging: vial

Expiration Date: 11-25-2014

Reason

Baxter Healthcare Corporation is voluntarily recalling lot LE12LG70AC of GAMMAGARD LIQUID, [Immune Globulin Intravenous (Human)] 10%, 20g size, product code 1502797, NDC# 0944-2700-06. This recall is being conducted as a precautionary measure due to a labeling error noted on the product vial label and unit carton. The labels on the unit carton and product vials indicate an incorrect manufacturing date and expiry date.

Incorrect Manufacturing Date: November 25, 2011
Incorrect Expiration Date: November 25, 2014

Correct Manufacturing Date: November 5, 2011
Correct Expiration Date: November 5, 2014

The Food and Drug Administration has been notified of this action.

Considering the information noted above, the medical assessment into this issue concluded that this labeling error poses negligible safety risk to the patient. This labeling error has no impact on the functionality of the product. Furthermore, stability data indicates the difference of 20 days between the correct expiry date and the printed expiry date has no impact on the product quality. Therefore, if you have already used this product no action is required. However, if you still have any inventory of this product lot, we are asking you to return the product.

Action

Please check all product labels against the affected lot number.
Do not take this product. Return affected recalled lots to the point of purchase to receive replacement product.

Other Information

If you need assistance, please call Stericycle, Inc. at 1-888-873-2838.

Call Gov. Cuomo & tell him to stand strong on passing a
NY Health Insurance Exchange in the final state budget!

As you all know, our State Senate is failing big time at responding to New York’s need for a health insurance Exchange. Up until now, they’ve been the only ones flip-flopping on this issue.Governor Cuomo, on the other hand, has been a champion from the get go on the push to get a health insurance Exchange bill passed in New York and even went so far as to put it in his State of the State address and in this year’s proposed state budget. But, now we’re getting reports that Gov. Cuomo is starting to waver on this issue. This is bad news and makes NOW a very opportune time to call Gov. Cuomo’s office to offer some much needed encouragement.

Call Gov. Cuomo at 518-474-8390.

Press # 3 to speak to an assistant, you will be asked for your zip code.

Press # 2 to leave a message with your idea for the future of New York.

Advocates Push for Better Insurance Coverage

for Children with Bleeding Disorders

Syracuse, N.Y. -- Bob Graham of Syracuse and other “bleeders” from around the state will travel to Albany Monday to ask legislators to fix what they say is a flaw in a state health insurance program that shortchanges children with hemophilia and other bleeding disorders.

Child Health Plus, a program for uninsured children in families with incomes too high to qualify for Medicaid, does not cover at home treatment for children with bleeding disorders such as hemophilia and Von Willebrand’s disease.

The state program only covers treatment if a child is admitted to a hospital. New York is the only state that does not cover at home treatments through its children’s health insurance program.

“Not adding this coverage is a question of fairness,” Graham said.

Bleeding disorders are among the most costly chronic diseases to treat. Annual treatment costs can exceed $100,000, according to the National Hemophilia Foundation.

People with these conditions often bleed longer than normal after an injury and can bleed internally, damaging organs. That’s because their blood does not clot normally.

Treatment involves infusions of blood clotting factor medicines that people can take at home. Before the advent of these medicines in the 1980s, people with bleeding disorders had to be hospitalized for treatment that involved whole blood and plasma tranfusions that were time consuming.

Graham and other members of the New York State Bleeding Disorders Coalition want legislators to pass a bill that would add at home coverage to Child Health Plus. Graham estimates there are about 50,000 state residents with bleeding disorders.

Assemblyman Micah Kellner, D-Manhattan, is sponsoring the bill in the Assembly. Sen. Joseph E. Robach, R-Rochester, is sponsoring the bill in the Senate. The measure was introduced last year, but never came up for a vote. Kellner said he hopes to get it passed this year.

Kellner said the coverage gap is an error that occurred when legislation was drafted 22 years ago creating Child Health Plus.

“It’s a mistake and we need to correct it,” Kellner said. “We have a moral obligation to provide that coverage.”

Bill proponents say providing treatment at home is less expensive than in a hospital. A bill memo says covering the at home treatments will be much less costly than the long term expenses likely to be incurred by Medicaid if these individuals develop debilitating complications and cannot work.

Child Health Plus is administered by the state Health Department. The program has not added any benefits that were not federally mandated since 2003 when hospice care was included. The department had no comment on the bill. A department spokesman said any change in coverage would have to be done by the Legislature.

Graham has hemophilia. His wife, Jessica, and stepson, Jeremy Kocsis, 15, both have Von Willebrand’s. Graham said his stepson is covered through his wife’s health insurance. Parents of children with bleeding disorders are routinely advised not to enroll their children in Child Health Plus because of the coverage gap, Graham said. If they don’t have employer-sponsored health insurance, these families typically enroll in Medicaid which covers in-home treatment, he said.

Parents that would like to earn more money and get off Medicaid often do not because their children will not be able to get at home coverage through Child Health Plus.

“It’s the standard of treatment for bleeding disorders,” he said.

Albany Days
March 18-19, 2012

This is your opportunity for you to learn about key legislative issues affecting the bleeding disorders community. It’s a chance for you to tell your stories to your elected representatives and staff members, helping them understand your needs and concerns.

TRAVEL and HOTEL expenses will be covered by HANY. If you are interested in going to Albany, please contact Annie at annhany@bestweb.net or 212-682-5510.

AGENDA:
Sunday, March 18
1:00PM – Registration
2:00PM – Advocacy Training
                     Followed by Dinner

Monday, March 19
All Day – Meet with your Representatives

Specific Issues and areas of concern:

S3865A CHP – non payment of factor in an outpatient setting.

S5849 Exchanges – ensuring that NY will have adequate coverage in their insurance exchanges scheduled to start coverage 2014.

Medicaid Redesign – currently factor is carved out of Medicaid Managed Care, but NYS Medicaid desires to have it covered under the Managed Care providers (not good for PHS or for profit providers).

Skilled Nursing Facility Access.

Group Meeting

March 15th

Thanks to everyone who attended.

For future meetings & updates, also go to our Facebook page:

www.facebook.com/Hemophilia.Association.of.NewYork

NHLBI to Provide $10M to

the Medical College of Wisconsin

The Medical College of Wisconsin has received a five-year, $10 million National Heart, Lung and Blood Institute Program Project Grant to continue genetic studies that seek to improve the molecular and clinical understanding of Von Willebrand disease (VWD), the most common hereditary bleeding disorder.

Von Willebrand disease is caused by a deficiency of von Willebrand factor (VWF), which is necessary for normal blood clotting. The disease affects both men and women. Most cases are mild, but aspirin and other nonsteroidal anti-inflammatory drugs can make the condition worse. Women may suffer very heavy menstrual bleeding, and family history of a bleeding disorder is the primary risk factor.

“There is a lack of understanding of the genetic causes of low or abnormal VWF, and the molecular mechanisms involved in the disorder,” said Dr. Montgomery. “While a large number of individuals have low VWF with abnormal bleeding symptoms, it is not scientifically clear if this is a disease, or if VWF is a continuous risk-factor for bleeding. For many practicing physicians, the general understanding of this group of disorders has not been optimal, and how to evaluate and treat these patients has been unclear.”

This study will determine the clinical and genetic characteristics of a large number of VWF patients as well as carriers of genes for various forms of the disease. The researchers will also explore the impact specific combinations of these genes and unrelated genetic mutations may have on individuals and families.

In addition, there are seven primary clinical centers (Milwaukee, Atlanta, Detroit, Pittsburgh, New Orleans, Indianapolis, and Iowa City) and more than 25 secondary clinical centers throughout the United States that recruit research subjects and send the samples to Milwaukee for the specialized testing. More than 600 families have been recruited for this project.

Washington Days
March 7 � March 9, 2012

Thank you to everyone who was representing HANY for Washington Days on March 7 – March 9, 2012.

If you were representing HANY in Washington D.C., we will reimbursed your TRAVEL and HOTEL expenses. Please SAVE ALL RECEIPTS and contact Annie at 212-682-5510 or annhany@bestweb.net for the reimbursement process. All meals were your responsibility.

Women's Group

February 16th, 2012

Thank you ladies for attending our
first women's group.

Help Support Our Mission with
Donations to the Annual Appeal

Don’t forget that there are people with bleeding disorders who need help.

Don’t forget that research to find a cure requires funding.

Don’t forget that we share a problem in common.

Don’t forget your contribution to the Annual Appeal.

Different ways to make a donation:

You can send us a check:
H.A.N.Y.
110 East 40th Street, Suite 506
New york, NY 10016

Use the link for PayPal:

Use the link for NYCharities:
www.nycharities.org/donate/c_donate.asp?CharityCode=3376

And don’t forget that donations to HANY are tax deductible.

6^th Annual Educational Symposium

Saturday, March 17^th, 2012

Grand Hyatt Hotel New York

8:30 am – 5:00 pm

Registration and exhibit registration forms online at
www.coalitionforhemophiliab.org

Contact: Kim Phelan 917-582-9077 or 212-520-8272

The Hemophilia Association of New York was founded in 1952.

Thanks to everyone who has supported us in continuing our mission to help
people with Hemophilia & other Bleeding Disorders.

We wanted to share the first edition of "The Bulletin",

which was our newsletter in 1952.

You can click on the link to read

www.hemophilia-newyork.org/images/The_Bulletin.pdf

Do you have Hemophilia?
Are you 18 or older?

Take an important survey

Resilience & Quality of Life in Individuals with Hemophilia

Funded by a Social Work Excellence Fellowship from
the National Hemophilia Foundation

Help us to learn more about the psychological and emotional
impact of aging with hemophilia, including the unique challenges you face,
how it affects your quality of life, and how you cope.　

The survey will take about 20 minutes to complete
and is completely anonymous. Go to:

www.surveymonkey.com/s/hemophilia248　　
or
www.henryford.com/body.cfm?id=40851
(click on "research study" in the middle of the page)

If you have questions, call:

                                                           Dr. Karen Allen
                                            Social Work ProgramAdult                                     　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　                          Oakland University,Rochester,Michigan
                                                  (248) 270-2502                                             　　　　　　　　　　　　　　　　　　　　　　　

     --- or ---
　　　　　　
       Ellen Kachalsky, LM
        HemophiliaTreatmentCenter
         HenryFordHospital,Detroit,Michigan　
    888-734-5322 or (313) 916-0470　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　                                　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　　

2012 DAY AT THE RACES
Help Support Our Fight
Against Hemophilia

Sunday, May 6, 2012 at 12:00 Noon
in Belmont Racetrack, Queens, NY

Contact Kathy Hornreich at 917-868-6958

FDA Approves Baxter's ADVATE as the Only FVIII for
Routine Prophylaxis in Both Adults and Children with Hemophilia A.

Click on link to read full article:
www.baxter.com/press_room/press_releases/2011/12_16_11_advate_prophylaxis.html

Men's Group
January 12th, 2012

Thank you gentlemen for attending our
men's group.

"Inhibitor Family Camp"
Comprehensive Health Education Services
Registration is currently open.

Comprehensive Health Education Services is pleased to present its� 3rd Inhibitor Family Camp weekend to be held on April 13-15, 2012 at The Painted Turtle Camp in Lake Hughes, CA.

This program is designed for families with a child age 6-19 with an active inhibitor. These children often report that they cannot participate in many of the activities offered at a traditional hemophilia camp due to the threat of injury. Or that if they do choose to participate, they may spend the rest of the week in a wheelchair watching from the sidelines. Inhibitor Family Camp provides these children and their families an opportunity to come together with their true peers in the hemophilia community because we believe that everyone deserves a camp to call their own.

Registration is currently open.

Space is limited so all registration materials must be completed in full by February 17, 2012. There is no cost to families associated with Inhibitor Family Camp. The program is generously supported by an educational grant from Novo Nordisk.

To register for camp, you must first complete the pre-registration form:
Website: www.comphealthed.com/IFC-Registration---April-2012.php

For additional information contact:
Comprehensive Health Education Services at 877-749-2437

"Inhibitor Family Camp" is sponsored by Comprehensive Health Education Services. HANY provides this material for your general information only. HANY does not engage in the practice of medicine, nor does it advocate or recommend specific medical treatments. You should consult your physician or local treatment center before attending.

Retailers Win on Pharma Bill

Despite a well-financed campaign by the pharmacy benefits management industry, Gov. Andrew Cuomo signed the controversial bill that bans health plans operating in New York from mandating that patients fill certain prescriptions by mail order. The measure was backed by New York's retail pharmacies but ran into heavy opposition from the PBM companies. A coalition that included the Medical Society of the State of New York promoted the bill (A-5502B) as pro-consumer because it gives plan members the option of getting their medications at either a pharmacy or by mail. It also means saving drugstore jobs. The PBMs charged that the ban would result in higher costs for health plans. A spokesman for PBM company Express Scripts said that the chapter amendments that will be added to the bill in January 2012 meant �the governor and Legislature clearly agreed with our position.� The governor signed the bill with the understanding that the chapter amendments will guarantee that retail pharmacies' reimbursement for a drug will not exceed that of mail-order suppliers. (From Crain's Health Pulse 12/14/11)

Hemophilia B Gene Therapy Breakthrough

Treatment for Blood Disease Is Gene Therapy Landmark

By Nicholas Wade
New York Times

Medical researchers in Britain have successfully treated six patients suffering from the blood-clotting disease known as hemophilia B by injecting them with the correct form of a defective gene, a landmark achievement in the troubled field of gene therapy. Hemophilia B, which was carried by Queen Victoria and affected most of the royal houses of Europe, is the first well-known disease to appear treatable by gene therapy, a technique with a 20-year record of almost unbroken failure.

�I think this is a terrific advance for the field,� said Dr. Ronald G. Crystal, a gene therapist at Weill Cornell Medical College. �After all the hype in the early 1990s, I think the field is really coming back now.� Gene therapy has had minor success in very rare diseases but suffered a major setback in 1999 with the death of a patient in a clinical trial at the University of Pennsylvania. Another gene therapy trial treated an immune deficiency but caused some cancer in soem patients.

The general concept of gene therapy � replacing the defective gene in any genetic disease with the intact version � has long been alluring. But carrying it out in practice, usually by loading the replacement gene onto a virus that introduces it into human cells, has been a struggle. The immune system is all too effective at killing the viruses before the genes can take effect. The success with hemophilia B, reported online Saturday in The New England Journal of Medicine, embodies several minor improvements developed over many years by different groups of researchers.

The delivery virus, carrying a good version of the human gene for the clotting agent known as Factor IX, was prepared by researchers at St. Jude Children's Research Hospital in Memphis. The patients had been recruited and treated with the virus in England by a team led by Dr. Amit C. Nathwani of University College London; researchers at the Children's Hospital of Philadelphia monitored their immune reactions.

Hemophilia B is caused by a defect in the gene for Factor IX. Fatal if untreated, the disease occurs almost only in men because the Factor IX gene lies on the X chromosome, of which men have only a single copy. Women who carry a defective gene on one X chromosome can compensate with the good copy on their other X chromosome, but they bequeath the defective copy to half their children. About one in 30,000 of newborn boys have the disease, with about 3,000 patients in the United States.

Dr. Nathwani and his team reported that they treated the patients by infusing the delivery virus into their veins. The virus homes in on the cells of the liver, and the gene it carries then churns out correct copies of Factor IX. A single injection enabled the patients to produce small amounts of Factor IX, enough that four of the six could stop the usual treatment, injections of Factor IX concentrate prepared from donated blood. The other two patients continued to need concentrate, but less frequently.

Treating a patient with concentrate costs $300,000 a year, with a possible lifetime cost of $20 million, but the single required injection of the new delivery virus costs just $30,000, Dr. Katherine P. Ponder of the Washington University School of Medicine in St. Louis notes in her commentary in The New England Journal of Medicine, calling the trial �a landmark study.�

The patients have continued to produce their own Factor IX for up to 22 months, said Dr. Edward G. D. Tuddenham, director of the Hemophilia Center at the Royal Free Hospital in London. One patient, a geologist, had a good response at first, but his level of Factor IX has declined to 1 percent of normal, the level at which the disease kicks in. �We attribute this to the fact that he had an inflammation, and although we treated it promptly, we should have been quicker off the mark,� Dr. Tuddenham said.

The patient cannot be injected again with the same virus because his immune system is now primed to attack it. �He�s very philosophic about it, but he�s a scientist, and his motivation is to help the science,� Dr. Tuddenham said. Twenty more patients will be treated to assess the best dose of the virus, the goal being the highest dose that does not set off an immune system attack, Dr. Tuddenham said. �We are pretty close to the sweet spot,� he said. If all goes well, a genetic treatment for hemophilia B �could be available for widespread use in a couple of years.�

In a trial in 2006, a patient injected with a corrective gene produced his own Factor IX but only for 10 weeks. The designer of that treatment, Dr. Katherine A. High of Children�s Hospital of Philadelphia, said the new therapy had worked because the delivery virus had been made more efficient and because the research team had treated the patients with steroids to suppress immune system attacks on the virus.

�I think it�s incredibly exciting, and I say that even though these people are my competitors,� she said. Dr. High is listed as a co-author of the report because her laboratory helped monitor the patients and provided proof for regulators that the virus would not insert its human gene into the patients� sperm and make the change hereditary. A serious problem with other delivery viruses is that they insert themselves randomly into chromosomes, sometimes disrupting a gene. The virus used by Dr. Nathwani�s team, known as adeno-associated virus-8, generally stays outside the chromosomes, so it should not present this problem. Still, patients will need to be monitored for liver cancer, a small possibility that has been observed in mice.

�I don�t think it�s a showstopper, but it�s a critical safety issue that has to be assessed,� Dr. High said. Patients have little or no immunity to the adeno-associated virus, which infects rhesus monkeys. The virus has a propensity for making liver cells its target, which is good for the therapy because these cells are the natural producers of Factor IX. However, liver cells do not live forever and slowly replenish themselves, possibly limiting how long the therapy will last.

About 80 percent of hemophilia cases are of the type known as hemophilia A, which is caused by defects in a different blood-clotting agent, Factor VIII. Researchers have focused on hemophilia B, in part, because the Factor IX gene is much smaller and easier to work with.

HEALTH-RELATED MATERIAL IN THIS NEWSLETTER IS PRESENTED FOR INFORMATIONAL PURPOSES ONLY. THE HEMOPHILIA ASSOCIATION OF NEW YORK (HANY) DOES NOT ENGAGE IN THE PRACTICE OF MEDICINE, NOR RECOMMEND SPECIFIC TREATMENTS OR DRUGS. YOU ARE URGED TO CONSULT YOUR PHYSICIAN OR LOCAL TREATMENT CENTER BEFORE PURSUING ANY COURSE OF TREATMENT. HANY DOES NOT ENGAGE IN THE PRACTICE OF LAW. SPECIFIC LEGAL ISSUES SHOULD BE DISCUSSED WITH A QUALIFIED ATTORNEY.

We'll have our first Women's Group in 2012!!!
We'll keep you posted.

Topics and contents of discussion � health related or not � is presented for informational purposes only. The hemophilia association of new york (hany) does not engage in the practice of medicine, nor recommend specific treatments or drugs. You are urged to consult your physician or local treatment center before pursuing any course of treatment. Hany does not engage in the practice of law. Specific legal issues should be discussed with a qualified attorney.

We have a new, easy & safe way for you to make a donation to us.
Remember that your credit card bill will indicate "NYcharities.org"
not our name because your donation is being processed by them.

Support our mission, this is the direct link:
www.nycharities.org/donate/c_donate.asp?CharityCode=3376

Thanks to all the men that were able to attend the group on November 17th. It was a very good informative session about health insurance. If you have any question about this, don't hesitate to call us!!!

Hope you enjoyed it!!!

If you weren't able to RSVP to attend to this meeting, check the mail, facebook & website for future meetings:
www.hemophilia-newyork.org/pastcurrentevents.html

Medical Advisory #415

Baxter Announces Labeling Error on Sterile Water

Baxter Healthcare Corporation has sent a notice of a labeling error affecting the expiration date of the Sterile Water for Injection that is packaged as a diluent with RECOMBINATE [Antihemophilic Factor (Recombinant)]. The Sterile Water for Injection label indicates that the shelf life is longer than what was actually approved by the FDA.

Please continue to use the RECOMBINATE product as labeled on the kit. There is no impact to the safety or efficacy of Recombinate if the Sterile Water for Injection diluent is used before the expiration date of the RECOMBINATE.

The following are the NDC and Lot numbers of the Sterile Water for Injection vials:

NDC Number | Lot Number | Size | Expiration Date
==========================================
0944284210 | TRA11802AC | 10 mL | 01-23-2014
0944284410 | TRA11810AC | 10 mL | 03-22-2014
0944284410 | TRA11803AC | 10 mL | 01-25-2014
0944284410 | TRA11803AD | 10 mL | 01-25-2014
0944284410 | TRA11810AA | 10 mL | 03-22-2014
0944284510 | TRA11805AA | 10 mL | 02-01-2014
0944284510 | TRA11805AB | 10 mL | 02-01-2014
0944284510 | TRA11805AC | 10 mL | 02-01-2014
0944284510 | TRA11819AA | 10 mL | 06-20-2014
0944284310 | TRA11808AC | 10 mL | 02-23-2014
0944284310 | TRA11816AA | 10 mL | 05-18-2014
0944284310 | TRB11820AA | 10 mL | 07-14-2014
0944284310 | TRA11806AC | 10 mL | 02-07-2014

PHYSICIANS: Please distribute this information to all providers in your area who treat patients with hemophilia.

CHAPTERS: Please distribute this information to your membership.

You must sign up for the Patient Notification System (PNS) to be notified directly about the latest recall or withdrawal of recombinant and plasma products. To enroll in the PNS, please call (888) UPDATE-U or go online at www.patientnotificationsystem.org

HANY provides this material for your general information only. HANY does not engage in the practice of medicine, nor does it advocate or recommend specific medical treatments. You should consult your physician or local treatment center before pursuing any course of treatment.

It's time to step up and let your voice be heard. The super committee is less than a week away to present their final plan to Congress to ensure both Medicaid and the Affordable Care Act are protected in the final deficit reduction package.

Take two minutes to call your members of Congress toll free today.

1. Call 1-888-876-6242 and ask the Capitol Hill Switchboard operator to be connected to one of your members of Congress.

2. Tell the staff member that you are calling to urge the member to reject any cuts to Medicaid as the super committee considers how to reduce the nation's deficit.

You can send an e-mail to your Senators and Congressmember using CIDNY's Legislative Action Center.

THANKS FOR YOUR SUPPORT!!!

NHF 63rd Annual Meeting

At NHF 63rd Annual Meeting with Linda, Joan, Annie, Ellen & Marta.

Thanks to the parents that were able to attend
the NY Presbyterian Support Group for Parents on
October 27th

Medical Advisory #414

Warning About Use of Non-Sterile Alcohol Wipes

In January 2011, NHF issued a Medical Advisory (#412) about a recall of non-sterile alcohol wipes produced by one company, Triad. In April, the FDA forced this company to close. Now, additional infections have occurred in patients using non-sterile alcohol wipes manufactured by another company, Pacific Disposables International (PDI). This company has also been required by the FDA to recall their products. Both of these companies package their products under their own label and under other companies� labels.

At this time, it appears prudent to recommend that all individuals who have alcohol wipes or other prep pads/swabs in their home examine the individual packages to see if they contain the word �Sterile.� This includes wipes that are packaged with factor or other IV preparation kits. If the word �sterile� does not appear on the package, there is no way to determine if they are in fact sterile or not. Therefore, individuals should discontinue use of any wipes that do not state �sterile� and request replacement with sterile wipes from their clotting factor distributor.

Nurses should also check their supply of alcohol wipes to ensure that they are only using sterile wipes. This is particularly important for patients with indwelling venous access devices (catheters or ports) and for patients with compromised immune systems, but should be standard of care for all patients.

PHYSICIANS: Please distribute this information to all providers in your area who treat patients with hemophilia. (October 26, 2011)

CHAPTERS: Please distribute this information to your membership.
You must sign up for the Patient Notification System (PNS) to be notified directly about the latest recall or withdrawal of recombinant and plasma products. To enroll in the PNS, please call (888) UPDATE-U or go online at www.patientnotificationsystem.org

HANY provides this material for your general information only. HANY does not engage in the practice of medicine, nor does it advocate or recommend specific medical treatments. You should consult your physician or local treatment center before pursuing any course of treatment.

On October 17, 2011, the New York Blood Center presented HANY with a check for $2,000. Thank you to all those who generously donated their �Red Cell and Platelet Advantage� points to the Hemophilia Association of New York. These people not only donate their blood cells to others, but have chosen to donate their Thank You gift to us.

For more information on this program go to: www.mydonoradvantage.com/choose.

From the New York Blood Center: Beth Shaz, MD, Vice President and Chief Medical Officer and Harvey Schaffler, Executive Director Donor Marketing;

From the Hemophilia Association of New York: Edward Rogoff, Board President and Linda Mugford, Executive Director.

Fall Family Retreat

Thanks to everyone who joined us in our first Family Retreat.
Check for updates for the 2012 camp invite.

Click on the link to see the group shot
www.hemophilia-newyork.org/campinformation.html

New York Medicaid
and
Family Health Plus (FHP)

Beneficiaries

The following information pertains to anyone who receives healthcare benefits from New York State Medicaid or Family Health Plus.

If you are covered by one of these programs, you should have received a letter by now from the New York State Department of Health (NYS DOH), which highlighted changes in Medicaid pharmacy services beginning October 1, 2011. Please be aware that these changes do not affect hemophilia clotting factor therapies. You can continue to receive your factor from your current specialty pharmacy provider (i.e., Hemophilia Treatment Center 340B program or other specialty pharmacy or home care company). These will continue to be covered as a Medicaid fee-for-service benefit. New York State is considering making changes to the way Medicaid and FHP cover factor in the future, but as of now factor is exempt from these changes.

Please note this does not replace previous advisories from the NYS DOH requiring you to select a Medicaid Managed Care (MMC) plan for your basic health care coverage. The MMC plan you chose will provide coverage for all Medicaid benefits except factor products. Examples of some of the services that will be covered by the MMC plan are preventive health and dental services, hospital inpatient and outpatient services, laboratory and x-ray services, and other prescriptions drugs.

If you have any questions, you may contact the New York State Department of Health Medicaid Helpline at 866-976-3526. You can also contact your Hemophilia Treatment Center or the Hemophilia Association of New York at 212-682-5510.

Back-to-school preparation is better done before the first day of school. But if you haven�t done so you are still in time to tell the school about your child�s bleeding disorder.

It�s important to contact the principal, school nurse and your child�s teachers (including gym instructors) to explain to them about the bleeding disorder your child has.

Make sure the school has your contact information up-to-date to get in touch with you in case of an emergency. Also provide them the contact information of your child�s doctor.

Your child should be able to participate in all of his classes, including physical education. However, if frequent bleeds happen during gym class, your doctor or the physical therapist at the HTC can work with the school to arrange a modified exercise program.

HANY can provide you with free pamphlets for the school, to explain hemophilia. Call us for copies at 212-682-5510. Your hemophilia treatment center is also available as a resource for starting the school year right.

It�s important to keep communication open between home and the school.

♦♦ Emergency Preparedness ♦♦

The National Hemophilia Foundation (NHF) has prepared a document called: At a Glance! Key Steps for Individual/Family Preparedness in an Emergency, click here to read it, which helps family members follow simple steps to prepare in case of an unexpected emergency.

If you are a residents of New York City, you can receive emergency notifications from the city's website by clicking here to register or by calling 311. All other residents should contact their local government office to find out the emergency plans in your area.

CAMP SESSIONS ARE CLOSED

We will keep you posted for 2012 camp sessions at any
of the three specialty camps nearest to our area.
All three camps provide on-site medical care and supervision.

              Hole in the Wall            Double H Ranch          Camp High Hopes
                  Ashford, CT.                     Lake Luzerne, NY.             Brantingham, NY.

Remember...

It is very important for you to register with the
Patient Notification System (PNS)
to inform you when a blood product
is withdrawn or recalled.

Click here to get more information
and to register at their website.

Medical Advisory #413

Baxter Voluntarily Recalls One Lot of Recombinate

Baxter BioScience has announced that it is voluntarily recalling one lot of Recombinate Antihemophilic Factor (Recombinant). Baxter states that this recall is being taken as a precautionary measure after a retrospective review of its manufacturing process revealed a breach in aseptic processing. This lot did meet all in-process and final container specifications, which include sterility and pyrogenicity. There have been no adverse events reported regarding use of this lot of Recombinate to date. This action is being taken with the knowledge of the U.S. Food and Drug Administration (FDA). (July 28, 2011)

Recalled Lot:

Lot Number - TRA09834AB
AHF IU/vial - 1060
Expiration Date 1-28-2012

If you have any of this product in your possession, please contact Baxter Customer Service at: 1-800-423-2090 for instructions on how to return the product and obtain a replacement.

You must sign up for the Patient Notification System (PNS) to be notified directly about the latest recall or withdrawal of recombinant and plasma products. To enroll in the PNS, please call (888) UPDATE-U or go online at www.patientnotificationsystem.org

HANY provides this material for your general information only. HANY does not engage in the practice of medicine, nor does it advocate or recommend specific medical treatments. You should consult your physician or local treatment center before pursuing any course of treatment.

New York: Tell Your State Legislators to Support Coverage for Outpatient Clotting Factor Products

Earlier this year Representative Kellner and Senator Robach introduced legislation, A6039A and S3865-A, on behalf of the New York State Hemophilia Advocacy Coalition. Passage of these pieces of legislation would allow the Child Health Plus (CHP) and Family Health Plus (FHP) programs to begin providing coverage for outpatient blood clotting factor products for children and adults with hemophilia and other bleeding disorders.

These public health insurance programs were designed to provide a way for low-income New Yorkers to access quality health care. In fact, New York is the only state not providing coverage for outpatient clotting factor products under the Children�s Health Insurance Program (CHIP). Passage of A06039A and S3865-A will rectify this unfair practice, ensuring that CHP and FHP eligible individuals are able to access necessary treatment.

We urgently need your help to get the bills passed before the legislative session ends. A06039A is currently before the Assembly Ways and Means committee. S3865-A is before the Senate Finance committee.

Please contact your State senator and representative TODAY and ask them to urge the Chairs of the respective committees above to put A06039A and S3865-A up for a vote.

Go to http://capwiz.com/hemophilia/issues/alert/?alertid=50700761 to take action, and you can deliver the letter by via email or print the letter to send it to your senator via USPS .

Thank you for your support.

On June 9th, we got together to honored Mr. Harrington's retirement at the Princeton Club. We thank you, Thom, for your 30+ years of your devoted work with the Association. Thanks to everyone who was able to join us in this celebration.

Check some of the pictures here:
www.hemophilia-newyork.org/pastcurrentevents.html

Our New Executive Director

The Hemophilia Association of New York welcomes Linda E. Mugford as our new Executive Director. Ms. Mugford holds degrees in Business Administration and Organizational Management, and has 25 years experience in not-for-profit and health-related careers.

As former manager of Comprehensive Hemophilia Services of New York Blood Services, Ms. Mugford not only brings knowledge of the issues facing those with bleeding disorders, but has a track record of action, working with both consumers and health care providers, and as an advocate for affordable access to medical treatment.