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

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, ...

- Following FDA pre-NDA feedback, submission on track for December 2024 - WARREN, N.J., Oct. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (PTCT) provided today several positive updates on the ...

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

Lexeo Therapeutics announced positive interim data for LX2006 in Phase 1/2 studies targeting Friedreich ataxia (FA) cardiomyopathy, indicating surpassing results in frataxin expression and left ...

BALA CYNWYD, Pa., Sept. 28, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare ...

Biogen Netherlands has received marketing authorisation from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for its Skyclarys (omaveloxolone) to treat Friedreich's ataxia in ...

Shares of Larimar Therapeutics rose in premarket trading after the company said it would share data from its study for its proposed treatment of Friedreich's ataxia, a neurodegenerative disorder, ...

Phase III trial successfully achieved its primary endpoint and key secondary endpoints with high statistical significance Levacetylleucine demonstrated clinically meaningful improvements in ...

Larimar Therapeutics, Inc. , is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. Larimar’s lead compound, nomlabofusp, is being developed as a ...