Companies in #Friedreich's Ataxia

Biogen is a leading global biotechnology company that discovers, develops, and delivers innovative therapies for serious neurological and neurodegenerative diseases. Its portfolio includes medicines for multiple sclerosis, spinal muscular atrophy, Alzheimer's disease, and rare diseases, with a growing late-stage neurology pipeline and recent expansion into rare diseases through the Reata Pharmaceuticals acquisition.

Therapeutics company focused on Friedreich's Ataxia. Received IND clearance for GV101 from the FDA in August 2026.

Minoryx Therapeutics is a clinical-stage biotechnology company developing differentiated therapies for X-linked adrenoleukodystrophy (X-ALD) and other orphan CNS diseases. Its lead candidate, leriglitazone, is an oral, brain-penetrant PPAR-γ agonist for cerebral ALD and Friedreich's ataxia.