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

Topline results show eDSP failed to significantly improve RmICARS scores in ataxia-telangiectasia patients compared with placebo in the NEAT trial.

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

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

Hereditary hemorrhagic telangiectasia (HHT) is characterized by extensive telangiectasias and arteriovenous malformations. The primary clinical manifestation is epistaxis that results in ...

Hereditary hemorrhagic telangiectasia (HHT) can cause recurrent, severe epistaxis, as well as anemia and reduced quality of life. The disease remains without licensed therapies worldwide. In this ...