SPINAL MUSCULAR ATROPHY

Clinical trials for SPINAL MUSCULAR ATROPHY explained in plain language.

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Trials to join now! 8 Not yet recruiting 1 Not yet finished but already full! 8 Completed 11 Terminated 1

Gene therapy trial targets kids with spinal muscular atrophy who can sit but not stand

Disease control Terminated

This study tested a gene therapy called AVXS-101 in children aged 6 months to 5 years with spinal muscular atrophy (SMA) who could sit but not stand or walk. The goal was to see if the treatment was safe and could help them gain motor skills like standing or walking. The trial wa…

Matched conditions: SPINAL MUSCULAR ATROPHY

Phase: PHASE1 • Sponsor: Novartis Gene Therapies • Aim: Disease control

Last updated May 15, 2026 11:54 UTC

SPINAL MUSCULAR ATROPHY

Get alerted when new SPINAL MUSCULAR ATROPHY trials appear

Gene therapy trial targets kids with spinal muscular atrophy who can sit but not stand