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News & Perspectives

Infusing Love: Meet Kristi

July 19, 2017

  Happy summer to all! I’m honored to join the ranks of the Infusing Love bloggers, and appreciate the opportunity to share my family’s stories and perspectives on living with hemophilia. Our family includes myself, my husband Nick, and our son, Liam, who is 4 ½. 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 “discussion draft” 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 “Surviving Voices” 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. Read the press release from Spark Therapeutics in it’s entirety here. Spark Therapeutics recently announced that 10 participants in its ongoing Phase 1/2 clinical trial of 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. Read the press release from BioMarin in it’s 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 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. Read the press release from uniQure in it’s entirety here. uniQure N.V. has announced updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy 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 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. Read the press release from Alnylam in it’s entirety here. Alnylam Pharmaceuticals, Inc. and Sanofi Genzyme, 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. 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 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. Read the press release from Genentech in it’s entirety 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®) 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 Eculizumab (Soliris®) in patients with the rare blood disorders, 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. Read the press release from Shire in it’s entirety here. Shire has announced 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 Therapeutics. Read the press release from Sigilon Therapeutics in it’s entirety here. Flagship Pioneering, a fully-integrated life science innovation enterprise, recently announced the launch of Sigilon Therapeutics Inc. Sigilon Therapeutics is a biopharmaceutical company that discovers and develops category-defining biocompatible encapsulated cell therapies. Sigilon Therapeutics’ 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’s easy to act first – 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’ve all been there – 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’Brittinee 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).  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 “discussion draft” 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 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 […]

Fatherhood & Hemophilia

June 18, 2017

Being a father has been the most challenging and most rewarding task God has ever asked of me. I would do it all over again in a heartbeat. I have found the relationship between my daughter, Bethany, and me to be such paradox at times, but now that we’re both adults, I can’t express how […]

Intern Introspective: Fly In Recap

June 16, 2017

Earlier this summer, we introduced you to La’Brittinee and Paul, our summer Policy interns. They have been busy all over DC, attending Congressional hearings and learning the ropes of working for a national organization. During their first week, they got to experience first-hand the planning and execution of our 4th Annual Patient Fly-In. Below are […]

Infusing Love: Wonder Women

June 14, 2017

  Motherhood is hard work, and often, moms don’t give themselves credit where credit is due. As moms, we need to brag about ourselves and celebrate our victories. We asked our Infusing Love bloggers to share those motherhood moments where they thought to themselves, “Yeah, I’m owning this mom thing.” Try to think of your […]

FDA Accepts Bioverativ’s Application for Investigational Hemophilia A Drug

June 12, 2017

Bioverativ announced on June 12, 2017 that the U.S. Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug (IND) application for BIVV001 (also known as rFVIIIFc-VWF-XTEN), a novel, investigational factor VIII therapy designed to potentially extend protection from bleeds with prophylaxis dosing of once weekly or longer for people with hemophilia A. […]

Dear Addy: History

June 12, 2017

Dear Addy, My daughter has informed me that most people in her generation don’t know anything about AIDS, Hemophilia, or Ryan White. I find this pretty upsetting. How can we educate the next generation about the history of the bleeding disorders community? Sincerely, Old Enough to Remember Dear Remembrance, This is a great question and […]


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