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., Kölbel 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., SAPPHIRE Study Group .

DOI

10.1080/14737175.2025.2579903

Type

Journal article

Publication Date

2026-01-01T00:00:00+00:00

Volume

26

Pages

101 - 120

Total pages

19

Keywords

Apitegromab, Motor, Motor neuron, Muscle, Myostatin, Neuromuscular, Plain language summary, Spinal Muscular Atrophy

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