MPR

Levacetylleucine Shows Promise for Treatment of Ataxia-Telangiectasia

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

Athena Diagnostics Improves Ataxia Movement Disorder Testing with Novel Long-Read Gene Sequencing

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 ...
News Medical on MSN

CIRM awards $7.4 million to advance stem cell-based gene therapy for Friedreich's ataxia

The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San Diego team developing a first-of-its-kind stem cell-based gene therapy for ...

Solid Biosciences Doses First Participant in First-in-Class Phase 1b FALCON Trial Evaluating SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

About the FALCON Trial FALCON is a first-in-human, open-label, multi-center Phase 1b clinical trial designed to evaluate the safety and tolerability of SGT-212 in participants aged 18-40 who have been ...