GENE THERAPY
Clinical trials for GENE THERAPY explained in plain language.
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One-Time gene therapy helps babies with rare 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 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 Jun 16, 2026 12:51 UTC
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Global study probes hemophilia b Patients' views on gene therapy
Knowledge-focused CompletedThis study interviewed 150 hemophilia B patients, their caregivers, and healthcare workers worldwide to learn what they know and think about gene therapy. The goal is to create better educational materials and improve the informed consent process, especially in countries with lim…
Matched conditions: GENE THERAPY
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 15, 2026 18:57 UTC