Donate

FDA Approves First 14-Day Hemophilia B Treatment

March 5, 2016

Note: The following is an edited form of a press release from the FDA . The original form of the release can be read聽here. The U.S. Food and Drug Administration approved on Friday, March 4, Idelvion, Coagulation Factor IX (Recombinant), Albumin Fusion Protein, for use in children and adults with Hemophilia B. Idelvion is the […]

HFA Observes February 29 As Rare Disease Day

February 23, 2016

The Hemophilia Federation of America (HFA) joins the National Organization for Rare Disorders (NORD) on February 29, and others around the world in observing Rare Disease Day. The purpose of this day is to focus attention on the needs of patients and families affected by rare diseases. In the U.S., any disease affecting fewer than […]

Dear Addy: Why Are Medical Alert IDs Important?

January 15, 2016

Dear Addy, Several parents that I鈥檝e met at the HTC have recommended I should get my daughter a medical ID bracelet. Why is this so important? Are there resources available to help me get her one? Signed Inquiring About IDs Dear Inquiring, For patients with serious medical conditions like a bleeding disorder, it鈥檚 important that […]

Dimension Therapeutics Announces Initiation of Phase 1/2 Study For Hemophilia B Treatment

January 7, 2016

Note: The below is an edited press release from Dimension Therapeutics, Inc. The original release can be read here. Dimension Therapeutics, Inc. announced on January 7 the initiation of a phase 1/2 study of DTX101, the company鈥檚 lead therapeutic candidate, for the treatment of patients with hemophilia B. DTX101 is designed to deliver stable expression […]

uniQure Announces Preliminary Results from Hemophilia B Gene Therapy Trial

January 7, 2016

Note: the following is the 聽edited form of a 聽press releaseoriginally published by uniQure. The full release can be read here.聽 On Thursday, January 7, 2016, uniQure N.V. announced preliminary topline results from the low-dose cohort of an ongoing Phase I/II clinical trial being conducted in adult hemophilia B patients treated with uniQure鈥檚 novel AAV5/FIX […]

Phase I Study Results for Once-Monthly Subcutaneous Hemophilia Treatment

December 8, 2015

Note: The following is an edited press release from Alnylam. The original release can be read in its entirety here. Alnylam Pharmaceuticals, Inc. announced on December 7, 2015 positive results from its ongoing Phase 1 clinical study with fitusiran (fi-TOO-si-ran), the recommended International Nonproprietary Name (INN) for ALN-AT3. Fitusiran is an investigational RNAi therapeutic targeting […]

New Data Released on Long-Acting Hemophilia B Treatment

December 8, 2015

Note: The following is the edited version of a press release issued by CSL Behring. The original release can be found here. On December 7, 2015, CSL Behring聽presented data from its Phase III PROLONG-9FP clinical program evaluating the efficacy and long-term safety of its investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant […]

Pain and Mobility Are Major Problems for Patients with Hemophilia

December 8, 2015

Note: The following originally appeared on MDMagazine.com. New research further supports the understanding that patients with hemophilia suffer from pain and mobility issues, and it went on to uncover which joints are most greatly affected by the condition. Christine Kempton, MD, MSc, from Emory University in Georgia, and colleagues used multiple strategies to find how […]

Dear Addy: Inhibitors

November 23, 2015

Dear Addy, I’m taking my son in for his annual visit, and I saw that MASAC issued a statement about inhibitors. Should I have him checked for one? Signed, Learning All I Can Dear Learning, Inhibitors are antibodies that the immune system develops in response to a clotting factor product used to treat a person […]

Dear Addy: Biosimilars

November 9, 2015

Dear Addy, I keep hearing about biosimilars. What are they, and how do they differ from generic drugs? Sincerely, Curious About Products Hi Curious, Thanks for your question. A biosimilar is a pharmaceutical drug that is made to have similar active properties as a biological drug that has already been licensed. Biologics are really important […]

New Study Open To Hemophilia B Community

September 24, 2015

Novo Nordisk, along with the Coalition for Hemophilia B, Hemophilia Federation of America, and National Hemophilia Foundation have announced the B-HERO-S (Bridging Hemophilia Experiences Results and Opportunities into Solutions) study open to all patients with hemophilia B and the caregivers of children with hemophilia B. About the Study Building on the success of the original […]

FDA Approves Fast Track Designation for New Hemophilia B Treatment

September 18, 2015

Note: This is an edited form of a press release from Dimension Therapeutics, Inc. To read the original release in its entirety,聽click here. Dimension Therapeutics, Inc. announced on Thursday, September 17 that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the company鈥檚 lead product candidate, DTX101, for the treatment of […]

New Data Supports Safety & Efficacy Claims of ALPROLIX

August 17, 2015

Note: This is an edited form of a press release from Biogen. To read the original release in its entirety, click here.聽 New clinical data support the long-term safety and efficacy of ALPROLIX庐 [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in people with severe hemophilia B treated for up to two years, Biogen announced on […]

FDA Approves New Hemophilia B Treatment

April 30, 2015

  Note: This is an edited version of a Emergent BioSolutions Inc.聽press release. To read the full release,聽click here. Emergent BioSolutions Inc. announced on Thursday, April 29 the聽U.S. Food and Drug Administration聽(FDA) has approved IXINITY庐, an intravenous recombinant human coagulation factor IX therapeutic for the control and prevention of bleeding episodes and for perioperative management […]

CSL Behring Renews Pledge to World Federation of Hemophilia

April 15, 2015

  In recognition of World Hemophilia Day April 17, the World Federation of Hemophilia (WFH) and CSL Behring announced today that CSL Behring has once again committed to donating bleeding disorder protein therapy international units (IUs) to the WFH Global Alliance for Progress (GAP) Program and other WFH programs, as well as making significant financial […]

Reuters: Britain’s Prime Minster Apologizes For Infected Blood In 70s, 80s

March 27, 2015

British Prime Minister David Cameron apologized on Wednesday, March 26, 2015 to thousands of people who were infected with HIV and Hepatitis C after being treated for unrelated conditions with contaminated blood in the 1970s and 1980s. Survivors and relatives of people who died after being infected through blood therapies or transfusions have campaigned for […]

Baxter Sees Promising Results in Inhibitor Treatment Trial

March 13, 2015

Baxter International Inc. announced positive results from its Phase III clinical trial evaluating the safety and efficacy of BAX 817, an investigational recombinant factor VIIa (rFVIIa) treatment for people with Hemophilia A or B who develop inhibitors. The prospective, open-label, randomized, multicenter trial was designed to assess the safety and efficacy of BAX 817 in […]

New Gene Therapy For Hemophilia Shows Potential As Safe Treatment

March 12, 2015

Research showed that bleeding events were drastically decreased in animals with Hemophilia B. Using a viral vector to swap out faulty genes proved safe and could be used for the more common Hemophilia A. _______________________________ A multi-year, ongoing study suggests that a new kind of gene therapy for Hemophilia B could be safe and effective […]

Infusing Love: Tough Questions to Answer

March 11, 2015

I was starting to get Ryan, my youngest, ready for bed while Logan hovered around us. Logan loves Ryan, and wants to be near him always. It is incredibly sweet. But on this particular day Logan was paying close attention to Ryan, and after a few moments Logan casually asked, 鈥淲hen does Baby Ryan get […]

Infusing Love: A True Black & Blue Sensation

March 4, 2015

While the rest of the world is debating whether a dress is black and blue or gold and white, one thing I know for certain is that my son, Thomas, has been black and blue many times over the last eleven years. Do you think the world understands why? You might remember HFA鈥檚 Hemophilia Awareness […]

Biogen Idec and Sobi See Progress in Pediatric Hemophilia B Study

February 27, 2015

This is an edited version of a press release from Biogen Idec. To read the full release, please聽click here. ____________________ Biogen Idec聽 and聽Swedish Orphan Biovitrum聽AB announced positive top-line results of the Kids B-LONG Phase 3 clinical study that evaluated the safety, efficacy and pharmacokinetics of ALPROLIX庐聽[Coagulation Factor IX (Recombinant), Fc Fusion Protein] in children under […]

Infusing Love: A Life Never Imagined

February 25, 2015

Have you ever signed up for something you felt you were not quite qualified for, but went ahead and did it anyway? I have, and I鈥檓 preparing to do it again.鈥 Several years ago, when I first heard about Washington Days, I never imagined myself actively engaging with legislators.聽 For most of my life, I […]

Walgreens Infusion Services Becoming a Private Independent Company

February 19, 2015

  Walgreen Co., a wholly owned subsidiary of Walgreens Boots Alliance, Inc. announced on January 20, 2015 that it has signed a definitive agreement with Madison Dearborn Partners (MDP), a leading private equity firm based in Chicago, under which MDP will acquire a majority interest in Walgreens Infusion Services. With more than 30 years of […]

Infusing Love: An Unexpected Haven

February 18, 2015

I鈥檇 be lying if I said my nerves weren’t through the roof upon entering the gates to the Taylor Family Foundation鈥檚 Camp Arroyo. To say that going to a family camp took me out of my element would be an understatement. I had no idea what I was getting myself into or what to expect. […]

Living Fit With von Willebrand Disease

February 13, 2015

One of the most common bleeding disorders in women is von Willebrand Disease (vWD), which occurs in about 1% of the US population. Heavy bleeding is one of the most common problems women report to their doctors. It affects more than 10 million American women each year. This means that about one out of every […]

Octapharma Launches Co-Pay Assistance Program

February 6, 2015

Octapharma USA announced on聽January 27, 2015 information about a new financial support program for the bleeding disorders community. The Octapharma Co- Pay Assistance Program is available to von Willebrand鈥檚 Disease (VWD) patients who are currently receiving wilate庐 [von Willebrand Factor/Coagulation Factor VIII Complex (Human)] or have a prescription to begin therapy. The new program offers […]

FDA To Review CSL Behring’s New Long-Lasting Hemophilia B Product

February 4, 2015

CSL Behring聽announced that the U.S. Food and Drug Administration (FDA) has accepted for review its Biologics License Application (BLA) for the marketing authorization of its long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). Upon FDA approval, rIX-FP will provide hemophilia B patients with a long-acting treatment option with dosing intervals up […]

Biogen Idec Forms Partnership to Focus on Gene Therapy for Hemophilia

January 30, 2015

Biogen Idec, Fondazione Telethon, and Ospedale San Raffaele have entered into a worldwide collaboration to jointly develop gene therapies for the treatment of both hemophilia A and B. The agreement will combine聽San Raffaele – Telethon Institute for Gene Therapy鈥檚 (TIGET)聽extensive expertise in creating new gene therapy strategies and developing them from the bench to bedside […]

Infusing Love: Hemophilia is the Easy Part

January 7, 2015

鈥淗emophilia is the easy part.鈥 I realized that both my son and I have said this recently.聽 There are many physical and emotional issues that we both face stemming from Max鈥檚 hemophilia, but the actual condition is not one of them. I鈥檒l say it again: 鈥淗emophilia is the easy part.鈥 I鈥檓 not even sure when […]

CSL Behring Submits FDA Approval for Long Acting Hemophilia B Product

December 16, 2014

CSL Behring announced on Tuesday, December 16, 2014 that it has submitted a biologics license application (BLA) to the United States Food and Drug Administration (FDA) for the marketing authorization of its long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). Once approved by the FDA, rIX-FP (Coagulation Factor IX {Recombinant}, Albumin […]


Sign up for E-mails, Dateline Magazine, and other ways to stay connected.