If approved, levacetylleucine could become the first therapy on the market for ataxia-telangiectasia.

Phase 3 trial results show levacetylleucine improves SARA scores in patients with the rare disorder ataxia-telangiectasia.

Please provide your email address to receive an email when new articles are posted on . Vatiquinone inhibits 15-Lipoxygenase, which regulates the pathways that Friedreich’s ataxia disrupts. PTC ...

LAS VEGAS, June 5, 2025 /PRNewswire/ -- DelveInsight's Friedreich's Ataxia Market Insights report includes a comprehensive understanding of current treatment practices, Friedreich's ataxia emerging ...

IntraBio Inc. today announced positive topline results from its pivotal Phase III IB1001-303 clinical trial, "Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Randomized, ...

A Prescription Drug User Fee Act target date of August 19, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) ...

SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations -- Dosing of the first participant in the Phase 1b ...

Spinocerebellar Ataxia (SCA) is a rare, genetic, life-threatening neurodegenerative disease with no available treatment. Troriluzole demonstrated a 50-70% slowing of SCA disease progression on the ...

The troriluzole clinical development program encompassed the first industry clinical trials to generate data showing therapeutic potential in patients with spinocerebellar ataxia (SCA), a rare genetic ...

Lexeo Therapeutics (NASDAQ:LXEO) added ~29% in the premarket on Tuesday after the company announced that the U.S. FDA has defined a pathway that will allow a potential accelerated approval for LX2006, ...

It looks like Biogen Inc.’s Nrf2 activator, Skyclarys (omaveloxolone), will maintain its status as the sole therapy approved for treating patients with Friedreich’s ataxia (FA), at least for now. The ...