About 30% of patients with periodic paralysis have unknown genetic mutation, making identification and treatment a challenge. Here: key points on genetics, testing, and subtypes of PPP.
Ulefnersen met its primary end point in the phase 3 FUSION trial, providing the first placebo-controlled evidence for a genetically targeted therapy in FUS-ALS.
The approval, based on phase 3 IB1001-303 trial data showing consistent SARA scale improvement, makes levacetylleucine the first treatment approved specifically for ataxia-telangiectasia.