论文信息 - P.371 Safety and efficacy findings in the first-in-human trial (FIH) of the oral splice modulator branaplam in type 1 spinal muscular atrophy (SMA): interim results - 字舞流文

P.371 Safety and efficacy findings in the first-in-human trial (FIH) of the oral splice modulator branaplam in type 1 spinal muscular atrophy (SMA): interim results

J. Praestgaard | E. Voltz | E. Bertini | N. Goemans | G. Baranello | N. Deconinck | U. Schara | L. Charnas | A. Hartmann | R. Roubenoff | A. Born | C. Pfister | T. Peters | C. Berghs-Clairmont | M. D. Raspide