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

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

Friedreich’s ataxia (FA) is an autosomal recessive disorder caused by mutations in the FXN gene and characterized by cardiomyopathy, gait ataxia and sensory loss, with cardiac complications the main ...

Ataxia-telangiectasia is an autosomal recessive disorder caused by a defect in the ATM gene mapped to chromosome 11q22.3. The ATM gene product is involved in the detection of damaged DNA and plays a ...

Researchers have discovered that extracts from plants used by the Kwakwaka'wakw First Nations peoples in their traditional botanical medicine practices are able to rescue the function of ion channel ...

BOSTON, July 29, 2025 /PRNewswire/ -- Athena Diagnostics, the specialty neurology business of Quest Diagnostics (NYSE: DGX), a leading provider of diagnostic information services, today announced a ...

Normal pressure hydrocephalus (NPH) is one of the few reversible causes of dementia in older adults and accounts for approximately 6% of all dementias. The cardinal sign of NPH is a hypokinetic gait ...