BioMarin Pharmaceutical Inc.
ABOUT
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with multiple commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
770 Lindaro Street
San Rafael, CA 94901
105 Digital Drive
Novato, CA 94949
San Rafael, CA 94901
105 Digital Drive
Novato, CA 94949
NEWS
Going through the most newsworthy stories of the year, BioSpace found trends more than one big story, topics that just kept rising again and again. Here’s a look.
BioMarin Pharmaceutical scored a first on Friday and provided an option for patients with a rare disease for which there is little recourse.
Mid-November is a relatively quiet period for the U.S. Food and Drug Administration in terms of its scheduled PDUFA dates. Read on for more.
QurAlis’ first piece of the puzzle is a protein called stathmin-2, which is encoded by the STMN2 gene.
It was a very busy week for clinical trial announcements, although there were only three that were COVID-19-related. Here’s a look.
Gene therapy clinical trials have been paused due to safety concerns, including the most recent one by BioMarin Pharmaceutical.
The oncogenicity and neurotoxicity risks of AAVs and possible mitigation strategies were the primary focus on Thursday.
The U.S. FDA had a very packed calendar for this week, but almost everything on it was under a Priority Review pathway.
Updated trial data show patients treated with BioMarin Pharmaceutical’s severe hemophilia A gene therapy expressed substantially lower factor VIII levels after five years compared with one year, calling into question the drug’s long-term benefits.
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