To evaluate the efficacy and safety of levacetylleucine in individuals diagnosed with Ataxia-Telangiectasia.
Conducted as a pivotal Phase 3 trial
Included both children and adults
Analyzed efficacy and safety results
Efficacy results demonstrate significant improvement in symptoms associated with Ataxia-Telangiectasia.
Safety profile remains consistent with previous findings, with manageable side effects.
Abstract
To report efficacy and safety results from the pivotal, Phase 3 trial investigating levacetylleucine in people living with Ataxia-Telangiectasia (A-T).