Voices of Friedreich's Ataxia (FA)

Significant advances in gene replacement therapy and the ability to edit genes have garnered much interest as a treatment approach

Nucleic acids are the building blocks of DNA and RNA. Oligonucleotides are chemically synthesized short DNA or RNA molecules that

Design Therapeutics is advancing a novel approach to activate transcription of the FXN gene and restore the production of frataxin

Resveratrol is a naturally occurring compound found in the skin of red grapes. Resveratrol has been under investigation as a

Etravirine (Brand name: Intelence) – Dr. Roberto Testi and his team at the University of Rome and Fratagene recently reported

Dr. Mark Payne, who first described the possibility of frataxin replacement therapy via TAT-Frataxin, has been developing this exciting prospect

Dr. Gino Cortopassi at the University of California Davis and other groups have observed that Nrf2 (Nuclear factor (erythroid-derived 2)-like

Elamipretide is a peptide compound developed by Stealth Therapeutics. It targets the inner mitochondrial membrane where it binds reversibly to

PTC-743 / Vatiquinone (previously EPI-743), is a follow-on or “sister” compound to EPI-A0001, which aims to improve mitochondrial and cellular

It is important to understand the burden of F A, including its extent and the most salient elements. The objective