Solid Biosciences Receives FDA Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations -- Dosing of the first participant in the Phase 1b ...

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 ...
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California Grants $7.4 Million to Advance Gene-Edited Stem Cell Therapy for Friedreich’s Ataxia

Funding supports safety studies, manufacturing and clinical planning needed before applying to test the treatment in patients ...
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What is Ataxia?

Ataxia is a form of movement disorder. There is loss of balance and physical co-ordination leading to difficulty in walking normally. The Cerebellum of the brain is normally responsible for ...