A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophy

Crawford TO, Servais L, Mercuri E, Kolbel H, Kuntz N, Finkel RS, Krueger J, Batley K, Dunaway Young S, Marantz JL, Song G, Yao B, Zhao G, Rossello J, Tirucherai GS, Mazzone ES, Butterfield RJ, Gomez Garcia de la Banda M, Seferian AM, Sansone VA, De Waele L, van der Pol WL, Cances C, Pechmann A, Darras BT

Expert Review of Neurotherapeutics