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 ...

A key opinion leader explores new and promising treatment approaches for managing advanced stages of Friedreich ataxia. In your opinion, what are the most promising emerging therapeutic avenues for ...

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 ...

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 ...

Friedreich’s ataxia is a chronic, progressive neurological condition that leads to impaired muscle coordination. This can affect balance and mobility as well as speech and heart health. Friedreich’s ...

A new gene therapy appears to be safe in patients diagnosed with Friedreich ataxia cardiomyopathy, a progressive and fatal inherited cardiac disease, according to a phase 1 clinical trial led by Weill ...

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 ...

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. Researchers ...

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 ...

The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San Diego team developing a first-of-its-kind stem cell-based gene therapy for ...