GENE THERAPY
Clinical trials for GENE THERAPY explained in plain language.
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Breakthrough 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 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 17, 2026 06:52 UTC
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Global survey reveals what hemophilia b patients really know about gene therapy
Knowledge-focused CompletedThis study interviewed 150 people with hemophilia B, their caregivers, and healthcare workers from around the world to find out what they know and think about gene therapy. The goal is to create better educational materials and improve the consent process, especially in countries…
Matched conditions: GENE THERAPY
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated May 17, 2026 06:47 UTC