GENE THERAPY
Clinical trials for GENE THERAPY explained in plain language.
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One-Time gene therapy helps babies with deadly muscle disease sit and breathe on their own
Disease control CompletedThis phase 3 trial tested a one-time gene therapy called Zolgensma in 22 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment delivers a working copy of the missing SMN gene via an IV infusion. The main goals were …
Matched conditions: GENE THERAPY
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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Global study probes patient views on gene therapy for hemophilia b
Knowledge-focused CompletedThis study interviewed 150 people with hemophilia B, their caregivers, and healthcare workers from around the world to learn what they know and how they feel about gene therapy. The goal is to create better educational materials and improve the consent process, especially in coun…
Matched conditions: GENE THERAPY
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:57 UTC