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News & Perspectives

Intern Introspective: That鈥檚 a Wrap for La鈥橞rittinee and Paul

July 28, 2017

Earlier this summer we welcomed our summer policy and government relations interns to our D.C. office. La鈥橞rittinee and Paul are part of the bleeding disorders community and were chosen to participate in a 10-week internship because of their leadership qualities and interest in the field of policy and advocacy. Throughout the summer, La鈥橞rittinee and Paul: […]

Infusing Love: Mama Is Always Right

July 28, 2017

  The early years of my motherhood experience were pretty lonely. We lived across the country from our extended family and I didn鈥檛 know any one in the neighborhood, and frankly, the other moms I did meet weren鈥檛 exactly people I wanted to socialize with. My go-to鈥檚 for parenting advice were books I had bought […]

ACTION ALERT: Vote Likely on Tuesday, July 25!

July 24, 2017

On Tuesday, the Senate will likely vote on a 鈥淢otion to Proceed,鈥 which is the first step to open debate on health reform legislation.聽 If the motion passes, they may consider a 2015 bill that will repeal parts of the Affordable Care Act without replacing it, or the Better Care Reconciliation Act (BCRA), the recent […]

Dear Addy: PRIDE Project

July 24, 2017

Dear Addy, I have heard of HFA鈥檚 PRIDE Project but am not clear on how I can participate and what I can learn from my participation. As a bleeding disorders community member, what do I need to know about the project and can I really do research? Signed, Researching Research Dear Researcher, You do research […]

Intern Introspective: Plasma Donation Center Tour

July 21, 2017

Nearing the end of their 10-week internship, La鈥橞rittinee and Paul got an opportunity to tour Grifol鈥檚 plasma collection center in Frederick, Maryland. Centers like this collect plasma from donors and then return the red blood cells to the donor in one sitting. The plasma, which undergoes several levels of strict testing, is then pooled and […]

Infusing Love: Meet Kristi

July 19, 2017

  Happy summer to all! I鈥檓 honored to join the ranks of the Infusing Love bloggers, and appreciate the opportunity to share my family鈥檚 stories and perspectives on living with hemophilia. Our family includes myself, my husband Nick, and our son, Liam, who is 4 陆. We live in Vermont right on the border with […]

Update 2: Bioverativ Files Complaints Against CSL Behring’s Idelvion

July 19, 2017

As noted in our July 10, 2017 story, Bioverativ has filed complaints in the U.S. District Court for the District of Delaware and with the U.S. International Trade Commission alleging that Idelvion, a novel factor IX albumin fusion protein developed by CSL Behring, infringes upon three Bioverativ patents. HFA, in collaboration with the National Hemophilia […]

ACTION ALERT: July 17, 2017 Update on BCRA

July 17, 2017

A vote on the health care bill will happen soon, and our organizations need you to take action. On July 13, 2017, the Senate released another 鈥渄iscussion draft鈥 of the BCRA. Despite promises from numerous lawmakers to change the bill, the latest iteration of the bill actually decreases protections for those with pre-existing conditions. The […]

FAQ: Hemophilia Memorial at the National Memorial AIDS Grove

July 12, 2017

  How did the Hemophilia Memorial at the National AIDS Memorial Grove come to be? In 2016, National AIDS Memorial Grove reached out asking for support for their 鈥淪urviving Voices鈥 project. This is a multi-year oral history project meant to ensure that stories and lessons of the AIDS epidemic are captured, curated, and retained for […]

Infusing Love: Not What I Had Planned

July 12, 2017

I like control. I like order. I like a plan. I like lists. Not in an overly aggressive way, but in a way that makes me feel calm. If I can plan for it, I can feel in control of it. Hemophilia does not work this way. Hemophilia and all the moving parts involved with […]

Spark Therapeutics Presents Updated Interim Hemophilia B Data Supporting Consistent & Sustained Response at ISTH 2017

July 11, 2017

Note: The following is an edited version of a press release from Spark Therapeutics.聽Read the press release from Spark Therapeutics聽in it鈥檚 entirety here. Spark Therapeutics recently announced that 10 participants in its ongoing Phase 1/2 clinical trial of聽SPK-9001 for hemophilia B, as of the June 5, 2017 data cut off, had their AIR reduced approximately […]

BioMarin’s Investigational Gene Therapy for Hemophilia A Maintains Average Factor VIII Levels within Normal Range for over One Year

July 11, 2017

Note: The following is an edited version of a press release from BioMarin.聽Read the press release from BioMarin聽in it鈥檚 entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. The updated results were聽presented […]

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

July 11, 2017

Note: The following is an edited version of a press release from uniQure.聽Read the press release from uniQure聽in it鈥檚 entirety here. uniQure N.V.聽has聽announced updated results聽from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy聽in patients with severe hemophilia B. The data includes up to 18 months of follow-up from the low-dose […]

Bioverativ Files Complaints Against CSL Behring’s Idelvion

July 10, 2017

Note: This story has been sourced from the website, www.thepharmaletter.com聽and The Australian Business Review. On Friday, July 7, 2017, Bioverativ filed complaints in the U.S. District Court for the District of Delaware and with the U.S. International Trade Commission alleging that Idelvion, a novel factor IX albumin fusion protein developed by CSL Behring, infringes upon […]

Alnylam & Sanofi Genzyme Report Positive Results from Ongoing Phase 2 Study with Investigational RNAi Therapeutic Fitusiran in Patients with Hemophilia A and B With or Without Inhibitors

July 10, 2017

Note: The following is an edited version of a press release from Alnylam.聽Read the press release from Alnylam聽in it鈥檚 entirety聽here. Alnylam Pharmaceuticals, Inc. and Sanofi Genzyme,聽announced today new positive results from the ongoing Phase 2 open-label extension study with fitusiran in patients with hemophilia A and B, with or without inhibitors.聽These results were presented today […]

Shire Gets Injunction Against Roche Over Emicizumab

July 10, 2017

Note: The following is an excerpt from a Reuters news article. Read the article in full here. Pharmaceutical group Shire said on Sunday it had obtained a preliminary injunction in a Hamburg court against rival Roche聽over its hemophilia drug emicizumab, alleging incomplete and misleading statements surrounding the treatment. Roche said in an emailed statement it […]

New England Journal of Medicine Publishes Articles in Conjunction with ISTH 2017

July 10, 2017

The New England Journal of Medicine has published two articles and an editioral in conjunction with the International Society of Thrombosis and Haemostasis meeting being held in Berlin, Germany, July 8-13, 2017. The articles include information on novel hemophilia treatment products currently in clinical trial. Click on the links below to read the articles in […]

Genentech Announces Positive Phase III Results for Emicizumab at ISTH 2017

July 10, 2017

Note: The following is an edited version of a press release from Genentech.聽Read the press release from Genentech聽in it鈥檚 entirety聽here. Genentech, a member of the Roche Group, announced today that data from HAVEN 1, a Phase III study evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) in adults and adolescents with hemophilia A with inhibitors, were published […]

Dear Addy: Hematology Visit

July 10, 2017

Dear Addy, I often become flustered when I visit my hematologist, despite having lots of questions. Can you offer guidance on how to prepare for my visit so I get my questions answered? Signed, Stressed by Appointment Dear Stressed, Any doctor visit can generate a case of nerves. If you have butterflies in your stomach […]

CDC Health Advisory: Patients Receiving Eculizumab (Soliris庐) at High Risk for Invasive Meningococcal Disease Despite Vaccination

July 7, 2017

Per an early release of the Centers for Disease Control and Prevention (CDC) Morbidity and Mortality Weekly Report (MMWR), new information was released today regarding聽Eculizumab (Soliris庐) in patients with the rare blood disorders,聽paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Read the MMWR article in full here.  

Shire Submits Investigational New Drug Application to FDA for Gene Therapy candidate SHP654 for treatment of Hemophilia A

July 7, 2017

Note: The following is an edited version of a press release from Shire.聽Read the press release from Shire聽in it鈥檚 entirety聽here. Shire has announced聽the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for SHP654, also designated as BAX 888, an investigational factor VIII (FVIII) gene therapy for the […]

New Biopharmaceutical Company, Sigilon Therapeutics, Launches

July 7, 2017

Note: The following is an edited version of a press release from Sigilon聽Therapeutics.聽Read the press release from Sigilon聽Therapeutics聽in it鈥檚 entirety聽here. Flagship Pioneering, a fully-integrated life science innovation聽enterprise, recently聽announced the launch of Sigilon Therapeutics Inc. Sigilon Therapeutics is a聽biopharmaceutical company that discovers and develops category-defining biocompatible encapsulated聽cell therapies. Sigilon Therapeutics鈥 discovery platform combines cell engineering and […]

FitFactor: Enjoying Summer

July 3, 2017

Summer is in full swing, and with so many fun things to do outside, it鈥檚 easy to act first 鈥 think later. But the hot summer months bring a certain level of risk, so here are a few precautions to take before heading outside. Heat and Sun: We鈥檝e all been there 鈥 we spend the […]

Intern Introspective: A Week in the Life

June 30, 2017

The halfway point of our Policy and Government Relations Internship program is an excellent time for the interns to reflect on the first five weeks of their internship. La鈥橞rittinee and Paul break down what an average week looks like for them as an HFA intern. Paul: After our first action packed week of the internship […]

Bleeding Disorders Organizations Urge Senators to Vote No on Better Care Reconciliation Act

June 29, 2017

  For the past few days, our organizations have been encouraging you to call, tweet and email your Senators to vote No on the Better Care Reconciliation Act (BCRA). 聽On June 27th, Senate Majority Leader Mitch McConnell delayed the Senate vote on the bill but the vote could occur quickly if Leader McConnell is able […]

Infusing Love: As He Climbs On the Bus

June 28, 2017

By now, for most of us another school year has come to an end. For me this usually means complete and utter joy! No more homework, no more early wakes up to catch the bus, no more worrying about clean PE uniforms, and the list goes on and on. Nicholas just finished the 8th grade. […]

Genentech Announces Results in Emicizumab Phase III Studies (HAVEN 1 And HAVEN 2) in Hemophilia A With Inhibitors

June 26, 2017

Genentech, a member of the Roche Group announced today positive data from the primary analysis of the Phase III HAVEN 1 study in adults and adolescents and interim analysis of the Phase III HAVEN 2 study in children evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) for the treatment of hemophilia A with inhibitors to factor VIII. […]

Dear Addy: Coverage for New Therapies

June 26, 2017

Dear Addy, I want to switch to a new hemophilia therapy and, though my doctor has prescribed the product, my insurer won’t authorize coverage. How can I get this covered? Signed, Concerned Consumer Dear Consumer, When new therapies come on the market, there is often a lag before health plans add those products to their […]

ACTION ALERT: Ask Your Senators to Vote NO on BCRA!

June 26, 2017

On Thursday, June 22, 2017, the Senate leadership released a 鈥渄iscussion draft鈥 of its version of legislation to repeal the Affordable Care Act (ACA). Despite promises from numerous lawmakers, the Senate bill in fact builds off the much criticized, House-passed American Health Care Act (AHCA). The Senate discussion draft would, among other things: Undermine聽protections for […]

Infusing Love: Change of Plans

June 21, 2017

  It’s that time of year when boys and girls go to summer camp. Church camp, art camp, band camp, sports camp and yes, hemophilia camps. My idea of camping and “roughing it” is when we stay at a hotel that is not a “suite.” When my oldest son, Julian, was nearing the age of […]


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