GENE THERAPY
Clinical trials for GENE THERAPY explained in plain language.
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Genom att skicka in godkänner du våra Användarvillkor
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One-Time gene therapy helps infants with devastating muscle disease sit and survive
Disease control CompletedThis study tested a single-dose gene therapy for babies under 6 months old with a severe, life-threatening genetic muscle disease called Spinal Muscular Atrophy (SMA) Type 1. The goal was to see if the one-time intravenous (IV) treatment could help infants achieve major milestone…
Matched conditions: GENE THERAPY
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Apr 04, 2026 00:21 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