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

The National Ataxia Foundation (NAF) is an organization devoted to raising awareness, supporting families, and funding research for Ataxia, a rare and progressive neurological condition that impairs ...

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

SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations - - Dosing of the first participant ...

Ataxia is a degenerative disease of the nervous system. Symptoms include slurred speech, stumbling, falling and incoordination. Find out what the National Ataxia Foundation is doing to fund research.

CHICAGO (WLS) -- The Friedrich's Ataxia Research Alliance (FARA) invites families, cyclists, and everyone else to their 5th Annual rideATAXIA Chicago event on July 24, 2016. The multi-route cycling ...

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 ...

RICHFIELD, Wis. (CBS 58) -- The 4th Annual National Ataxia Bowl for a Cure will take place Sunday, Sept. 28 at Pioneer Bowl in Richfield from 11 a.m. to 4 p.m. The fundraiser supports research, ...

Initial data from the Phase 1b FALCON trial expected in H2 2026, subject to participant enrollmentCHARLESTOWN, Mass., Jan. 12 ...

The Gehr family has always had a love and a talent for the performing arts. “I really lived a pretty unaffected life until about third grade,” said 25-year-old Michael Gehr. As a kid, Michael was what ...

The National Ataxia Foundation (NAF) is an organization devoted to raising awareness, supporting families, and funding research for Ataxia, a rare and progressive neurological condition that impairs ...