GENE THERAPY
Clinical trials for GENE THERAPY explained in plain language.
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One-Time gene infusion helps babies with fatal muscle disease sit up and survive
⭐️ CURE ⭐️ CompletedThis study tested a single-dose gene therapy for babies under 6 months old with spinal muscular atrophy type 1, a severe genetic muscle-wasting disease. The goal was to see if a one-time intravenous infusion could help babies achieve major milestones like sitting independently an…
Matched conditions: GENE THERAPY
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: ⭐️ CURE ⭐️
Last updated Mar 30, 2026 14:29 UTC
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Patients and doctors share views on new gene therapy for bleeding disorder
Knowledge-focused CompletedThis study surveyed 150 hemophilia B patients, their caregivers, and healthcare workers worldwide to understand what they know and think about gene therapy. Researchers conducted interviews to learn about people's attitudes, concerns, and how they prefer to receive information ab…
Matched conditions: GENE THERAPY
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:28 UTC