(BPT) – A rare disease is a disease, disorder, illness or condition that affects fewer than 200,000 Americans. 1 Since rare diseases impact a small number of people, they often remain relatively ...
DENVER — Four studies presented at the American Academy of Neurology annual meeting show improvements in patients with spinocerebellar ataxia who take troriluzole. “SCAs [spinocerebellar ataxias] are, ...
Cure Rare Disease (CRD), a clinical-stage nonprofit biotechnology company developing genetic medicines for ultra-rare and rare conditions, today announced that the U.S. Food and Drug Administration ...
Spinocerebellar ataxias are a group of neurodegenerative diseases characterized by the degeneration of Purkinje cells, a major class of neurons in the cerebellum. The resulting cerebellar dysfunction ...
- Spinocerebellar Ataxia (SCA) is a rare, genetic, life-threatening neurodegenerative disease with no available treatment. - Troriluzole has been granted Fast-Track, Orphan Drug Designation (ODD) and ...
European Commission approves OMP Designation for NLX-112 (befiradol) following compelling results in studies funded by the US Dept of Defense. This OMP designation is a milestone for Neurolixis as we ...
A new study finds enhanced activity of a specific enzyme drives spinocerebellar ataxia type 14, which suggests inhibiting the enzyme may have therapeutic potential. pinocerebellar ataxias are a group ...
The FDA has issued a CRL to Biohaven regarding the NDA for troriluzole for the treatment of adults with spinocerebellar ataxia.
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