Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced the results of a comprehensive investigation into the case of hepatocellular carcinoma (HCC) diagnosed in one patient in the HOPE-B pivotal trial […]
The following is an excerpt from a press release from uniQure. Read the update in its entirety聽here. Today uniQure announced a clinical hold by the U.S. Food and Drug Administration (FDA) in its聽phase III HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. Per uniQure鈥檚 statement: 鈥淭he clinical hold was initiated following […]
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure,聽a gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the late-breaking presentation of initial data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five […]
The following is from a press release from Pfizer. Read it in its entirety here. Pfizer Inc. announced that the first participant has been dosed in the Phase 3 BASIS study of marstacimab (PF-06741086), an anti-tissue factor pathway inhibitor (anti-TFPI) being evaluated for the treatment of people with severe hemophilia A or B, with or […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V.,聽a gene therapy company advancing transformative therapies for patients with severe medical needs, announced positive top-line data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based […]
CSL Behring issued the following statement to patient advocacy groups: “CSL Behring is focused on delivering breakthrough therapies to improve patients鈥 lives and meet the changing needs of our patients. Since CSL Behring鈥檚 introduction of MONONINE庐 Coagulation Factor IX (Human) in 1992, advancements have been made for the treatment of Hemophilia B.聽 Over time, patients […]
The following is an excerpt from a press release from Novo Nordisk. Read the press release in its entirety here. Novo Nordisk announced the clinical trials in the concizumab phase 3 programme (explorer 6, 7 and 8) are being resumed. The clinical trials are investigating subcutaneous concizumab prophylaxis treatment in hemophilia A and B patients […]
The following is an announcement from the Centers for Disease Control and Prevention. Hemophilia is an inherited bleeding disorder in which the blood does not clot properly due to a lack or decrease in a protein called clotting factor. The two most common types of hemophilia are hemophilia A, which is due to a lack […]
Novo Nordisk recently distributed a notice relevant to its product Rebinyn庐,聽raising awareness that one-stage clotting assay results can be significantly affected by the type of activated partial thromboplastin time (aPTT) reagent used, which can result in over or under-estimation of Factor IX activity. The notice which was addressed to HTCs, physicians and other healthcare providers, […]
The following is an excerpt from a press release from uniQure. Read the release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, reported recent progress across its business. 鈥淒espite the ongoing global crisis caused by the coronavirus pandemic, we ended the first quarter of […]
The following is a press release from HEMA Biologics. HEMA BiologicsTM Announces FDA Approval of SEVENFACT庐 [coagulation factor VIIa (recombinant)-jncw] for Treatment and Control of Bleeding Episodes Occurring in Adult and Adolescent Hemophilia A and B Patients with Inhibitors Louisville, KY 鈥 April 6, 2020 HEMA Biologics, LLC, (鈥淗EMA Biologics鈥) today announced that the U.S. […]
The following is a press release from uniQure. uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has achieved the targeted dosing of patients in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the […]
The following is an excerpt from a press release from Aptevo Therapeutics. Read the press release in its entirety here. Aptevo Therapeutics Inc. , a biotechnology company focused on developing novel immuno-oncology therapeutics based on its proprietary ADAPTIR™ bispecific technology platform, announced it has completed the sale of its marketed recombinant factor IX therapeutic, IXINITY庐 […]
Bullying has no shame. It shows no prejudice. Bullying affects anyone of any size, age, and ethnicity.聽 It affects all of us. When I watch grown people intentionally hurt each other with words and actions, it always surprises me. One day I asked a young lady why she was making fun of her coworker. […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated clinical data on the three patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene […]
Our son, Michael, recently surprised us with the news that he was going to get a tattoo! I know, I know, he is 26 years old and can make his own decisions.聽But he still has severe hemophilia B with inhibitors. I got that knot in my stomach that all moms get 鈥 it鈥檚 a […]
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure聽announced updated clinical data in patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant, for the treatment of patients with severe and moderately severe hemophilia B. […]
The following is a press release from Sanofi Genzyme. Find the full press release here. Sanofi Genzyme, the specialty care business unit of Sanofi, announced the launch of a first-of-its-kind audio adventure series designed to provide an engaging and educational infusion experience for ALPROLIX庐 [Coagulation Factor IX (Recombinant), Fc Fusion Protein] patients and their caregivers. […]
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure,聽a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced updated clinical data in patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a […]
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the […]
The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here. Hemophilia B is a rare, genetic bleeding disorder caused by a lack of the factor IX (FIX) protein, which is necessary for normal blood clotting. Current hemophilia B treatments typically require frequent intravenous infusions of clotting […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated results聽from its ongoing Phase I/II trial of AMT-060, and provided an update on AMT-061, the company鈥檚 next-generation gene therapy聽candidate […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure announces the initial topline data from our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B. All patients achieved and sustained therapeutic […]
The following is an excerpt from a press release from uniQure. uniQure announced聽that they have treated the first patient in our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B (see attached press release). 鈥淎s a one-time administered therapy, AMT-061 has the […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here.聽 uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy […]
The following is a summary of a press release from Catalyst Biosciences by Dr. Robert Sidonio, a trusted medical advisor. Read the full press release here. On June 18, Catalyst Biosciences provided an update of the progress from its Phase 陆 trial investigating the use of FIX variant product, called CB 2679d/ISU304, which is a […]
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 […]
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 […]
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 […]
Note: The followed is excerpted from a press release from uniQure.聽Read uniQure’s full press release HERE.聽 uniQure announced today聽plans to advance their development program for hemophilia B gene therapy into a pivotal study using an enhanced version of their聽product candidate called AMT-061. AMT-061 is identical to AMT-060 in every way apart from a change from […]
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