Friedreich's Ataxia Market Research and Forecast Report 2025-2035: Gene Therapy and Small-Molecule Innovations Redefine Treatment Paradigms

The global Friedreich’s ataxia market is experiencing notable growth, fueled by advancements in genetic research, enhanced diagnostic technologies, and increased awareness of this rare ...
The American Journal of Managed Care

Managing Advanced Stages of Friedreich’s Ataxia: Challenges and Emerging Therapeutic Avenues

A health care provider outlines treatment objectives and supportive interventions for managing Friedreich ataxia, addressing associated comorbidities and symptoms in clinical settings. August 7th ...
The American Journal of Managed Care

Managing Adverse Events in Novel Friedreich’s Ataxia Therapy

A key opinion leader outlines common adverse events associated with omaveloxolone treatment in Friedreich's ataxia patients and describes strategies used to manage or minimize these adverse effects.
Nasdaq

Solid Biosciences' SGT-212 Gene Therapy for Friedreich's Ataxia Receives FDA IND Clearance, Phase 1b Trial Set for 2025

Solid Biosciences announces FDA clearance for SGT-212, targeting Friedreich’s ataxia with dual gene therapy administration routes, initiating Phase 1b trial in 2025. Solid Biosciences Inc. announced ...
News Medical

Gene therapy used to overcome the cardiac effects of Freidreich’s ataxia

Gene therapy was successfully used to overcome the cardiac effects of Freidreich’s ataxia (FA) in a mouse model of the disease, as reported in the peer-reviewed journal Human Gene Therapy. Click here ...
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 ...
Healio

First patient dosed in phase 1/2a trial of Huntington’s, spinocerebellar ataxia therapy

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. The trial will evaluate VO659, an antisense ...

Lexeo Therapeutics: Behind The Rebound

Lexeo Therapeutics, Inc. surges after FDA trial alignment for LX2006 in Friedreich’s ataxia. Click for more on LXEO stock following its Q3 earnings release.
BioWorld

Lexeo, FDA position Friedreich ataxia therapy for accelerated path

Lexeo Therapeutics Inc. feels like it’s in a faster lane to a BLA for its Friedreich ataxia cardiomyopathy gene therapy after talking with the U.S. FDA. The agency told Lexeo that LX-2006 could be on ...
Business Wire

Papillon Therapeutics Receives Orphan Drug 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 ...
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 ...