Add Yahoo as a preferred source to see more of our stories on Google. Difficulty balancing or an inability to do so can be caused by any number of factors. Head injuries, migraines, alcohol use, ear ...

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

As IntraBio works to grow the global treatment market for Aqneursa in Niemann-Pick disease type C, the company is already ...

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

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the ...

(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 ...

Having a rare genetic disease is actually pretty common. Rare diseases affect approximately 1 in 10 individuals, and more than 30 million people in the U.S. have a rare disease diagnosis. What makes ...

Having a rare genetic disease is actually pretty common. Rare diseases affect approximately 1 in 10 individuals, and more than 30 million people in the U.S. have a rare disease diagnosis. What makes ...

~ Neurocrine Biosciences to pay Voyager Therapeutics $165 million upfront + up to $1.7 billion in potential milestone payments for collaboration in Parkinson’s disease and Friedreich’s ataxia ~ ...

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 ...