If approved, levacetylleucine could become the first therapy on the market for ataxia-telangiectasia.

Phase 3 trial results show levacetylleucine improves SARA scores in patients with the rare disorder ataxia-telangiectasia.

Several months after forging a fresh path forward for its troubled Friedreich ataxia (FA) drug vatiquinone, PTC Therapeutics is back with some much-needed positive data. The candidate passed muster by ...

Difficulty balancing or an inability to do so can be caused by any number of factors. Head injuries, migraines, alcohol use, ear infections and medications can all affect your musculoskeletal system ...

The FDA has granted orphan drug designation to Cure Rare Disease’s CRD-002, an antisense oligonucleotide therapeutic for the treatment of spinocerebellar ataxia (SCA), including spinocerebellar ataxia ...

Truist has initiated coverage of Larimar Therapeutics (NASDAQ:LRMR) with a buy rating, citing an "asymmetric" risk/reward ahead of regulatory updates for its drug candidate nomla for Friedreich’s ...

The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San ...

SAN DIEGO--(BUSINESS WIRE)--Papillon Therapeutics Inc., a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited ...