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.
The American Journal of Managed Care

Guidelines & FDA Approval of Novel Friedreich Ataxia Therapy

A key opinion leader examines the November 2022 guidelines for managing Friedreich's ataxia, discusses the first FDA-approved therapy for the condition, and explains how this treatment is integrated ...
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 ...
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 ...
MedCity News

Reata Pharma Drug Wins First FDA Nod in Ultra-Rare Neuromuscular Disorder

Friedrich’s ataxia, an ultra-rare disease characterized by progressively worsening muscle function that ultimately leads to death, now has its first FDA-approved therapy. The regulator has given the ...
Healio

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

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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 ...
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 ...
Insider Monkey

Lexeo Therapeutics’ Friedreich Ataxia Gene Therapy Receives FDA Breakthrough Therapy Designation

Lexeo Therapeutics Inc. (NASDAQ:LXEO) is one of the best IPO stocks to buy according to Wall Street analysts. Earlier on July 7, the US FDA granted Breakthrough Therapy designation to Lexeo ...
Business Wire

Cure Rare Disease Awarded $5.69 Million CIRM Grant to Advance Gene Therapy for Spinocerebellar Ataxia Type 3

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the ...
Seeking Alpha

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 ...