Seeking Alpha

Solid Biosciences Receives FDA Rare Pediatric Disease Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia

CHARLESTOWN, Mass., Dec. 01, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and ...
Business Wire

Papillon Therapeutics Receives Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich's Ataxia

SAN DIEGO--(BUSINESS WIRE)--Papillon Therapeutics Inc., a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited ...
Insider Monkey

Biogen Launches Phase 3 Pediatric Trial Of Omaveloxolone For Friedreich Ataxia.

Biogen Inc. (NASDAQ:BIIB) is among the 11 Best Genomics Stocks to Buy According to Hedge Funds. It has begun dosing in the global Phase 3 BRAVE trial to test omaveloxolone (SKYCLARYS®) in children ...
Nasdaq

Biogen Initiates Global Phase 3 BRAVE Study to Evaluate Omaveloxolone for Pediatric Friedreich Ataxia

Biogen Inc. has launched the BRAVE study, a global Phase 3 clinical trial aimed at assessing the efficacy and safety of omaveloxolone in children aged 2 to under 16 years with Friedreich ataxia (FA), ...
MPR

NEAT Trial: No Significant Improvement With eDSP in Ataxia-Telangiectasia

Topline results show eDSP failed to significantly improve RmICARS scores in ataxia-telangiectasia patients compared with placebo in the NEAT trial.
Business Insider

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 FALCON trial has been completed, with initial data ...