Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
Update for Hemophilia Associations BioMarin is pleased to update the community regarding our gene therapy clinical trial program in hemophilia A. BioMarin鈥檚 investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. BioMarin Hemophilia Update For Patient Associations View PDF | Download […]
BioMarin is pleased to update the community regarding our gene therapy clinical trial program in hemophilia A. BioMarin鈥檚 investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. Clinical Trial Overview BioMarin鈥檚 valoctocogene roxaparvovec, is currently being studied in adults with severe […]
The following is an excerpt from a press release from BioMarin. Read the press release in its entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. The data have been submitted […]
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 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 […]
The following is a press release from BioMarin. 聽BioMarin is pleased to update the community regarding our gene therapy clinical trial program in hemophilia A.聽 Clinical Trial Update聽 BioMarin鈥檚 investigational gene therapy valoctocogene roxaparvovec, is currently being studied in adults with severe hemophilia A.聽 The first Phase 1/2 study was initiated in 2015 and consists […]
The following is an excerpt from a press release from BioMarin. Read the press release in its entirety here. BioMarin Pharmaceutical Inc. announced its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A achieved pre-specified clinical criteria for regulatory review in the U.S. and Europe. As of May 28, 2019, eight patients in […]
The following is an excerpt from a press release from BioMarin. Read the entire press release here. BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The updated results were presented during […]
Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The data […]
Note: The following is an edited version of a press release from BioMarin.聽Read the press release from BioMarin聽in it鈥檚 entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. The updated results were聽presented […]
Note: The below is an edited version of a press release issued by BioMarin Pharmaceutical Inc. The original release can be read in its entirety聽here. BioMarin Pharmaceutical announced today positive interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. Nine patients with severe hemophilia […]
Note: The below is an edited version of a press release by BioMarin Pharmaceutical Inc. The original release can be read in its entirety here. BioMarin Pharmaceutical Inc. announced on Tuesday, March 1, that BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by […]
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