GENE THERAPY

Clinical trials for GENE THERAPY explained in plain language.

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Trials to join now! 2 Completed 1

One-Time gene therapy helps babies with fatal muscle disease sit and survive

Disease control Completed

This study tested a one-time gene therapy (AVXS-101) in 22 infants under 6 months old with spinal muscular atrophy 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 needing p…

Matched conditions: GENE THERAPY

Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control

Last updated May 01, 2026 15:57 UTC

GENE THERAPY

Get alerted when new GENE THERAPY trials appear

One-Time gene therapy helps babies with fatal muscle disease sit and survive