In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. These are defined as conditions affecting under 200,000 Americans.…
Recently, research and development (R&D) company Neurophth Therapeutics announced that the FDA granted Orphan Drug designation to its therapy NR082. Also known as rAAV2-ND4 or NFS-01 project, NR082 is…
After three years, patients with Leber hereditary optic neuropathy (LHON) who had been treated with LUMEVOQ experienced sustained safety and efficacy. According to a press release from GenSight Biologics,…
About BridgeBio Pharma BridgeBio Pharmaceuticals was created in 2015 by veterans from the fields of academia and biotechnology. The company focuses on the development of new therapeutic treatments for genetic…
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