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 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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

Friedreich's ataxia (FRDA) is a progressive neurodegenerative disorder caused by GAA repeat expansions in the frataxin (FXN) gene, leading to neurological dysfunction, muscle weakness, and ...

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 ...