SPINAL MUSCULAR ATROPHY

Clinical trials for SPINAL MUSCULAR ATROPHY explained in plain language.

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Trials to join now! 10 Not yet recruiting 1 Not yet finished but already full! 10 Completed 14 Terminated 1

Gene therapy zolgensma tested in kids with SMA who can sit but not stand

Disease control Terminated

This phase 1 trial tested a gene therapy called AVXS-101 (Zolgensma) in 32 children with spinal muscular atrophy (SMA) who could sit but not stand or walk. The therapy delivers a working SMN gene via a spinal injection to help improve muscle function. The study focused on safety …

Matched conditions: SPINAL MUSCULAR ATROPHY

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

Last updated Jun 27, 2026 08:14 UTC

SPINAL MUSCULAR ATROPHY

Get alerted when new SPINAL MUSCULAR ATROPHY trials appear

Gene therapy zolgensma tested in kids with SMA who can sit but not stand