Pandolfo M. Friedreich Ataxia: An (Almost) 30-Year History After Gene Discovery

Pandolfo M. Friedreich Ataxia: An (Almost) 30-Year History After Gene Discovery. Neurol Genet. 2025 Jan 13;11(1):e200236. doi: 10.1212/NXG.0000000000200236. PMID: 39810753; PMCID: PMC11731367. 

 Now, 28 years after the gene discovery, although much remains to be understood about the disease's mechanisms and the development of effective therapies, the progress is undeniable. A thriving community has emerged, uniting researchers, health care providers, industry professionals, individuals with FRDA, their families, and dedicated volunteers. With this collective effort, a cure is within reach.