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 2 Not yet finished but already full! 6 Completed 10 Terminated 1

One-Shot gene therapy tested in toddlers with devastating muscle disease

Disease control Terminated

This study tested a one-time gene therapy injection for children aged 6 months to 5 years with a specific type of spinal muscular atrophy (SMA). The main goal was to find a safe dose and see if the treatment could help children gain motor skills, like standing or walking. The tri…

Matched conditions: SPINAL MUSCULAR ATROPHY

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

Last updated Mar 27, 2026 12:38 UTC

SPINAL MUSCULAR ATROPHY

Get alerted when new SPINAL MUSCULAR ATROPHY trials appear

One-Shot gene therapy tested in toddlers with devastating muscle disease