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 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 […]
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 information is provided by BioMarin to provide general information about its ongoing clinical trials in the context of the COVID-19 pandemic.聽 “As more cases of COVID-19 are confirmed, we recognize the increased challenges and concerns faced by participants in the BioMarin Gene Therapy clinical studies. The safety and well-being of patients, healthcare providers, […]
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 announcement from BioMarin. BioMarin announced an update to the community regarding our ongoing gene therapy聽clinical trial program in hemophilia A. BioMarin鈥檚 investigational gene therapy for聽hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating聽its safety and efficacy. Clinical Trial Overview BioMarin鈥檚 investigational gene therapy valoctocogene roxaparvovec […]
Note: The below is an edited version of a press release by UniQure.聽The original release can be read in it’s entirety聽here. On June 11, 2016,聽uniQure N.V. announced that additional data from its Phase I/II clinical trial of AMT-060 in hemophilia B patients were presented at the 21st聽Congress of the European Hematology Association (EHA) in Copenhagen, […]
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