Nasdaq

Solid Biosciences Receives FDA Fast Track Designation for SGT-212, a Dual Route Gene Therapy for Friedreich’s Ataxia

Solid Biosciences' SGT-212, dual-route gene therapy for Friedreich’s ataxia, receives FDA Fast Track designation for expedited development. Solid Biosciences Inc. has announced that its gene therapy ...
EurekAlert!

Findings point to path forward for treatment of rare genetic disease

Friedreich’s ataxia (FA) is a rare but devastating genetic disorder. Those with the condition are often diagnosed between 5 and 15 years of age and live only into their 30s or 40s. There is no widely ...
Seeking Alpha

Larimar Therapeutics Receives FDA Clearance to Proceed to 50 mg Cohort in CTI-1601’s Phase 2 Friedreich's Ataxia Trial and to Initiate Open Label Extension Trial

Top-line safety, pharmacokinetic, and pharmacodynamic (frataxin level) data from Phase 2 trial’s 50 mg cohort expected in 1H 2024 Initiation of open label extension trial with 25 mg daily dosing ...
Morningstar

Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia

- Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track designation - CHARLESTOWN, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Solid ...