Lila Levinson, PhD,  Science Writer—

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to LX2006, a gene therapy for cardiomyopathy in Friedreich’s Ataxia (FA). The status means the Lexeo Therapeutics candidate may be eligible for fast track review and extra FDA guidance during the regulatory process, which could…

Biogen has initiated dosing in a Phase 3 clinical trial, called BRAVE, that’s testing the oral therapy Skyclarys (omaveloxolone) among children with Friedreich’s ataxia (FA) ages 2 to 15. Skyclarys already is approved in the U.S. and the European Union, among other locations, for FA patients ages 16…

A new gene editing technique disrupted the mutations that cause Friedreich’s ataxia (FA) in a mouse model, a study reports. A type of base editing, the technique swaps one genetic building block, or nucleotide, for another. These edits can interrupt long repetitions of three-letter DNA sequences (trinucleotide repeats, or…