Novartis Gene Therapies
Clinical trials sponsored by Novartis Gene Therapies, explained in plain language.
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Gene therapy breakthrough: one dose may help babies with rare muscle disease
Disease control CompletedThis study tested a single dose of a gene therapy called AVXS-101 in 30 infants under 6 weeks old who were genetically diagnosed with spinal muscular atrophy (SMA) but not yet showing symptoms. The goal was to see if the treatment could help them reach motor milestones like sitti…
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated May 26, 2026 07:16 UTC
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One-Time gene therapy helps babies with rare muscle disease sit on their own
Disease control CompletedThis study tested a one-time gene therapy (AVXS-101) in infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The main goal was to see if treated babies could sit without support for at least 10 seconds by 18 months of age. The t…
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated May 26, 2026 07:12 UTC
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One-Time gene therapy helps babies with fatal muscle disease sit and survive
Disease control CompletedThis study tested a one-time gene therapy (AVXS-101) in 22 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The goal was to see if the treatment helped babies sit independently for at least 30 seconds and survive without nee…
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated May 22, 2026 14:01 UTC
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One-Time gene therapy helps babies with rare muscle disease sit and breathe on their own
Disease control CompletedThis study tested a one-time gene therapy in 33 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment delivers a working copy of the missing SMN1 gene to help nerve cells survive. Results showed many babies could si…
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated May 22, 2026 13:50 UTC