Novartis Gene Therapies

Clinical trials sponsored by Novartis Gene Therapies, explained in plain language.

Now part of Novartis AG NVS NYSE

A subsidiary of Novartis focused on gene therapies.

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Completed 4 Terminated 1

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

Disease control Stopped early

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 …

Phase 1 • Sponsor: Novartis Gene Therapies • Aim: Disease control

Last updated Jun 27, 2026 08:14 UTC

Novartis Gene Therapies

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