BioMarin Releases Results of Phase 1/2 Study of Gene Therapy Treatment

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 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 A […]

Sangamo BioSciences Announces New Gene Therapy Clinical Development Program

Note: The below is an edited version of an article originally published on PR Newswire. The original release can be read in its entirety here.Sangamo BioSciences, Inc. announced the presentation of preclinical data that supports the clinical development of its new proprietary gene therapy for the treatment of hemophilia A. This new therapeutic comprises an adeno-associated virus […]

uniQure Presents Updated Clinical Data in Patients with Severe Hemophilia B

Note: The below is an edited version of an article originally published on Globe Newswire. The original release can be read in its entirety here.uniQure N.V. yesterday announced updated clinical data from its ongoing Phase I/II trial of AMT-060, its proprietary, investigational gene therapy, in patients with severe hemophilia B.  The updated data show that all […]

Hepatitis: Not Just For Men

I have severe factor 1 deficiency (afibrinogenemia) and have been Hepatitis C (HCV) positive since 1998. My source of infection was one of my many cryoprecipitate infusions (cryo being the only available treatment for anyone with factor 1 deficiency until 2009, and for me until November 2014). What makes me different than most people with […]