Differential (Click to cross reference) asymptomatic efficacy motor neuron disease muscle weakness preclinical prognosis progressive neurologic disorder risdiplam safety spinal muscular atrophy term infant treatment of neurologic disorder Werdnig-Hoffman disease

Showing articles 0 to 1 of 1 Risdiplam in Presymptomatic Spinal Muscular Atrophy NEJM 393:671-682, Finkel,R.S.,et al, 2025 Showing articles 0 to 1 of 1