SUNRISE-FA 2 study parameters include LVMI primary endpoint, 6-month topline efficacy analysis, inclusion criteria focused on ...

A health care provider outlines treatment objectives and supportive interventions for managing Friedreich ataxia, addressing associated comorbidities and symptoms in clinical settings. A key opinion ...

Goldenrod Therapeutics, innovator in brain inflammation treatments for neurological conditions, completed initial close on $6 ...

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.

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 ...

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. Click here ...

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 ...