We use cookies to provide you with a better experience. By continuing to browse the site you are agreeing to our use of cookies in accordance with our Cookie Policy.
Bluebird Bio’s gene therapy Skysona (elivaldogene autotemcel) has snagged accelerated approval as a treatment for early, active cerebral adrenoleukodystrophy (CALD), an invariably fatal genetic disorder that strikes young boys. Read More
An FDA advisory committee surprised observers on Wednesday night, voting 7-2 to endorse Amylyx Pharmaceuticals’ amyotrophic lateral sclerosis (ALS) drug despite FDA doubts and the committee’s own rejection of the drug six months earlier. Read More
The FDA isn’t impressed by the data Amylyx Pharmaceuticals is relying on in its second attempt to get the nod for AMX0035, its investigational amyotrophic lateral sclerosis (ALS) therapy. Read More
The company’s goal is to offer reduced dose frequency compared to immediate-release carbidopa/levodopa, the gold standard for treatment since the 1970s. Read More