Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
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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