Hemophilia Federation of America is a national nonprofit organization that assists, educates, and advocates for the bleeding disorders community.
On June 28, 2016, the National Hemophilia Foundation’s Medical and Scientific Advisory Committee (MASAC) issued a recommendation following the published results of the Survey of Inhibitors in Plasma Products Exposed Toddler (SIPPET) The SIPPET study was published in the New England Journal Of Medicine (NEJM) in May 2016. The full text of the SIPPET study is available for a […]
On May 26 2016, the New England Journal Of Medicine (NEJM) published the results of the Survey of Inhibitors in Plasma Products Exposed Toddler (SIPPET) study. Investigators found that in a randomized study, when previously-untreated patients (PUPs) were treated with recombinant factor VIII, there was an 87% higher incidence of inhibitor development than when treated with plasma-derived factor VIII. […]
Note: The below is an edited version of a press release by CSL Behring. The original release can be read in it’s entirety here. KING OF PRUSSIA, Pa., May 26, 2016 — CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has approved AFSTYLA [Antihemophilic Factor (Recombinant), Single Chain], its novel long-lasting recombinant factor […]
Note: the following is the edited form of a press releaseoriginally published by uniQure. The full release can be read here. 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’s novel AAV5/FIX […]
Note: The following is an edited press release from Octapharma USA. The original release can be read in its entirety here. Octapharma USA announced on Monday, January 4, 2016 that NUWIQ®, Antihemophilic Factor (Recombinant), is now commercially available. NUWIQ® is indicated for the treatment and control of bleeding, perioperative (surgical) management, and routine prophylaxis to reduce […]
This past weekend, the American Society of Hematology (ASH), a worldwide organization of 15,000+ medical and scientific experts on blood diseases, held their 57th Annual Meeting to discuss new treatments and research methods. During this meeting, a group of doctors from around the globe presented the findings of the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET). […]
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 […]
Micah will be 14 years old in just a couple of months. He has severe hemophilia A, no inhibitors, and has been on prophylaxis since he was just over a year and a half old. So, for roughly twelve years, he has been on a very strict prophylaxis schedule dosing three or four times a […]
Note: The below is an edited press release from CSL Behring. To read the full release, click here. CSL Behring announced that the U.S. Food and Drug Administration has accepted for review the company’s Biologics License Application (BLA) for its novel investigational recombinant factor VIII single-chain (rVIII-SingleChain) for the treatment of hemophilia A. In the […]
Note: This is an edited form of a press release from Baxalta, Inc., originally published on July 16, 2015. To read the full press release, click here. Baxalta Incorporated announced the publication of the complete data from the Phase II/III pivotal study and Phase I trial of BAX 855 in Blood, the journal of the […]
This is an edited version of a press release from Biogen Idec. To read the full release, please click here. ____________________ Biogen Idec and Swedish Orphan Biovitrum AB announced positive top-line results of the Kids B-LONG Phase 3 clinical study that evaluated the safety, efficacy and pharmacokinetics of ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in children under […]
This is an edited version of a press release from CSL Behring. To read the full release, please click here. ____________________ CSL Behring 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 […]
Although it is fact that my son, Christian, has severe hemophilia, and a fact that we had no family history, the treatment for his condition is not as simple a fact. I appreciate my ability to have a choice and voice in his treatment options, but sometimes I feel even more fear that I will […]
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