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News & Perspectives

Novo Nordisk Launches Rebinyn in the United States for People with Hemophilia B

February 21, 2018

Note: The following is an excerpt from a press release from Novo Nordisk. Read the full press release here.聽 Novo Nordisk, a global healthcare company, today announced that Rebinyn, Coagulation Factor IX (Recombinant), GlycoPEGylated, is now available in the United States for the treatment of hemophilia B. Rebinyn聽is an extended half-life injectable medicine used to […]

Dear Addy: Bullied for Bruising

February 20, 2018

  Dear Addy, My eleven-year-old son recently shared that he鈥檚 being bullied at school because of bruising caused by his bleeding disorder. How can I help him educate his classmates about hemophilia and prevent future bullying? As much as I want to protect my son, I also know that I need to empower him to […]

Infusing Love: Am I a Carrier?

February 14, 2018

Does life ever make sense? Do we really know the answers to why things actually happen? Losing my daughter, Sydney, nine days shy of her second birthday due to SUDC (Sudden Unexplained Death in a Child), pretty much means there is no answer to why. But my family decided to welcome another child into the […]

FitFactor: Functional Training – Strengthen Joints and Muscles to Improve Everyday Movements

February 5, 2018

Functional training exercises train your muscles to work together and prepare you for daily tasks by simulating common movements you might do at home, at work or in sports. By using basic movement patterns like pushing, pulling, squatting and carrying, you build stability, strength and mobility that will in turn help with your daily movements. […]

Dear Addy: Legislative Day Toolkit

February 5, 2018

Dear Addy, I鈥檓 participating in an Advocacy Day with my local bleeding disorders organization this month. I have limited experience with legislative advocacy and it鈥檚 been a long time since I took a civics class! Could you share insight on what to expect and how to prepare? Signed, Proactive Participant Dear Participant, This is a […]

Breakthrough in Hemophilia Gene Therapy

January 31, 2018

The following is an excerpt from an article in Medical News Bulletin. Read the full article here. Researchers at Spark Therapeutics and Pfizer tested the safety and efficacy of a new hemophilia gene therapy vector for the treatment of hemophilia B. The body鈥檚 ability to stop bleeding and repair damaged blood vessels depends heavily upon […]

Octapharma Support Enables Bombardier to Climb Mount Vinson, Becoming First Hemophiliac to Ascend the Seven Summits

January 24, 2018

Note: The following is an excerpt from a press release from Octapharma USA. Read the full press release here. Octapharma Partnership with Mountain Climber will Culminate with Release of Documentary Bombardier Blood Later This Year Mountain climber Chris Bombardier of Denver, Co., became the first hemophiliac to climb the Seven Summits of the world on […]

Infusing Love: Relating to 鈥榃onder鈥

January 24, 2018

When I committed to writing this week鈥檚 blog, I had no idea what I would blog about until I went to see the movie 鈥榃onder.鈥 Wonder is about a boy named Auggie who was born with a facial disease called聽mandibulofacial dystosis, also known as 鈥淭reacher Collins syndrome.鈥 Due to Auggie鈥檚 medical needs, which require the […]

Hemophilia Puts Damper on Patients鈥 Sex Life and Health Status, Study Reports

January 23, 2018

Study Shows Sexual Intimacy Correlates With More Bleeds The following is an excerpt from Hemophilia News Today. Read the entire article here. Researchers report that聽hemophilia聽interferes with patients鈥 sexual activity and is linked to worse health status. The study titled 鈥淪exual Health in Patients with Hemophilia; The Insights from the Patient Reported Outcomes, Burdens and Experiences […]

Sanofi to Acquire Bioverativ for $11.6 Billion

January 22, 2018

Note:聽The following is edited from a press release from Sanofi. Read the full press release in its entirety聽here. Sanofi and Bioverativ Inc., a biopharmaceutical company focused on therapies for hemophilia and other rare blood disorders, have entered into a definitive agreement under which Sanofi will acquire all of the outstanding shares of Bioverativ for $105 […]

Third Circuit Says Health Care Company’s Donations Didn’t Violate Anti-Kickback Statute

January 20, 2018

Note: The following information comes from an article published in the New Jersey Law Journal. Read the full article here. The U.S. Court of Appeals for the Third Circuit has cleared Medco Health Solutions of violating the Anti-Kickback Statute, finding in a precedential ruling that a whistleblower failed to show a connection between alleged kickbacks […]

Dear Addy: Project CALLS 2018

January 19, 2018

Dear Addy, I’m already experiencing problems with my insurance in the new year. Where can I go to report these issues? Signed, New Year, Old Issues Dear New Year, You are not alone. HFA regularly hears from community members with concerns about access to care due to limitations or mandates set by their insurance company. […]

Infusing Love: Ayudando Nuestras Familias

January 16, 2018

Puerto Rico, Isla del encanto, conocida por sus bellas playas, gente amigable y amable, en donde se baila la mejor salsa y se degustan los mejores sabores caribe帽os. Arropada por el Mar Caribe y el Oc茅ano Atl谩ntico. En donde se habla espa帽ol y es un territorio ligado a Estados Unidos como Estado Libre Asociado y […]

Consolidated Postings Regarding Shire’s Lawsuit Against Genentech/Roche

January 16, 2018

In an effort to provide the hemophilia community a centralized location to find the most up-to-date statements and press releases regarding a recent injunction Shire is seeking in its lawsuit against Genentech/Roche, HFA will publish links on this page. As a reminder,聽HFA will NOT engage in the dialogue around patent ownership and will not be […]

Shire Provides Statement Regarding Lawsuit

January 16, 2018

Shire has published a聽statement regarding the ongoing lawsuit Shire has filed against Genentech/Roche. Read the full statement聽in its entirety聽here.    

HFA and NHF Issue Joint Statement Regarding Shire Lawsuit Against Genentech/Roche

January 12, 2018

In the past few weeks, we have received many inquiries regarding the scope of the injunction Shire is seeking in its lawsuit against Genentech/Roche.聽 Certain information has become public during this period that enables NHF and HFA to provide some limited guidance as to the scope of the injunction that Shire has requested from the […]

Genentech Provides Statement Regarding Patient Access to Hemlibra

January 11, 2018

Genentech has published a聽statement regarding patient access to Hemlibra (Emicizumab-kkwh). Read the full statement聽in its entirety聽here.  

Fit Factor: Inspiring Fitness Stories to Get You Going in 2018

January 9, 2018

Below, we鈥檒l introduce you to three such people: Trevor Dunn, a young man with von Willebrand Disease, Dawn Evans, an adult woman who is asymptomatic carrier of Hemophilia A, and Barry Haarde, an adult male with severe Hemophilia A.聽 All of them have discovered a form of physical activity that is enjoyable and adaptable to […]

PSI Files Lawsuit Against U.S. Department of Health and Human Services

January 9, 2018

On January 9th, Patient Services, Inc. (PSI) filed a lawsuit against the Federal Government alleging that a 2017 Modified Advisory Opinion from the Office of the Inspector General (OIG) violates their First Amendment rights. For more information, please see the press release from PSI.

Alnylam and Sanofi Enter into Strategic Restructuring of RNAi Therapeutics Rare Disease Alliance

January 9, 2018

Note:聽The following is edited from a press release from Alnylam. Read the full press release in its entirety聽here. Alnylam Pharmaceuticals, Inc.,聽an RNAi therapeutics company, and Sanofi announced today a strategic restructuring of their RNAi therapeutics alliance to streamline and optimize development, and commercialization of certain products for the treatment of rare genetic diseases. Specifically, Alnylam […]

Dear Addy: Accumulator Adjuster Programs

January 8, 2018

Dear Addy, I get assistance with my out-of-pocket costs from the manufacturer of my clotting factor product. My insurer just notified me that it will no longer apply that assistance to my deductible and out-of-pocket maximums! Can they do that? Signed, Distressed Over Deductibles Dear Deductibles, You are running up against a new strategy that […]

Catalyst Biosciences Initiates Phase 2 of Potential Hemophilia A and B Therapy

January 5, 2018

Note:聽The following is聽an excerpt聽from an article by Rare Disease Report. Read the full聽article in its entirety聽here. On Jan. 4, Catalyst Biosciences announced the initiation and open enrollment of the Phase 2 part of its Phase 2/3 program of marzeptacog alfa (activated) (MarzAA), a highly potent, subcutaneously administered Factor VIIa therapy in development for the treatment […]

Infusing Love: Port to Adulthood

January 3, 2018

I have seen signs for the last few years, but on a cold day just before Christmas, I saw my 14-year-old son not as a little boy, but as a young man. There wasn鈥檛 anything extraordinary happening in the moment. He sat at one end of our sectional couch and I was sitting on the […]

Infusing Love: Different Opinions Don鈥檛 Mean They Are Wrong

December 27, 2017

鈥淪o, he鈥檚 still wearing the helmet?鈥 The question, posed by HTC staff at our recent annual visit gave me pause. Yes, my son is almost 5 years old and still wearing his helmet any time his feet are on the floor. I know this is a controversial topic 颅 I鈥檝e seen the comment threads on […]

Dear Addy: Public Speaking

December 25, 2017

Dear Addy, I want to participate in a legislative day, but I’m a terrible public speaker. Help! Signed, Nervous Nelly Dear Nervous, You are not alone 鈥 many people worry about public speaking! Use these tips to help you become more confident and comfortable speaking during legislative meetings. Hopefully you鈥檒l find this advice beneficial 鈥 […]

HFA Research Presents at ASH 2017

December 21, 2017

HFA鈥檚 Research Team gave and received early an holiday gift this year: knowledge. We attended the 59th annual meeting of the American Society of Hematology (ASH) in Atlanta, Georgia, December 9-12. The unofficial song of the meeting was 鈥淟et it Snow鈥 as that (along with the once standing Georgia Dome) is what was on the […]

Infusing Love: In Appreciation of Providers

December 20, 2017

It is safe to say that most of hemophilia has been unexpected. Even when you are planning for the unexpected you get another unexpected in its place, sort of a Murphy鈥檚 Law type deal. And yet, I am always finding things about hemophilia that I never expected from our experience. One of the most profound […]

Fit Factor: Spice-Up Your Holiday Dessert Menu

December 19, 2017

Looking for a new healthy holiday dessert? This Applesauce Spice Cake has a delicious, spicy aroma and taste. It has less sugar and fat, and more fiber than most desserts. Treat it like a coffee cake to be served for brunch or for dessert with a scoop of vanilla ice cream or a dab of […]

uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

December 15, 2017

  Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. uniQure N.V.聽a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced on Dec. 11, 2017, updated results聽from its ongoing, dose-ranging Phase I/II trial of AMT-060, its investigational gene […]

BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

December 15, 2017

Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The data […]


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