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Spinal muscular atrophy type 2
Clinical trials for Spinal muscular atrophy type 2 explained in plain language.
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One-Shot gene therapy aims to restore movement in children with SMA
Cure By invitation onlyResearchers test GCB-001, a gene therapy given as a single injection into the spinal fluid, in children aged 2 to 12 with type 2 spinal muscular atrophy who can sit alone but cannot walk. The trial checks safety and whether the treatment improves motor skills over 12 months. It i…
Matched conditions: SPINAL MUSCULAR ATROPHY TYPE 2
Sponsor: Genecombio Ltd. • Aim: Cure
Last updated Sep 13, 2026 00:00 UTC
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Can a muscle-boosting antibody help people with spinal muscular atrophy over the long haul?
Disease control OngoingThis trial tests the long-term safety and effectiveness of apitegromab, an investigational antibody that blocks myostatin, a protein that limits muscle growth. People with type 2 or type 3 spinal muscular atrophy who completed earlier apitegromab studies can join. Researchers wil…
Matched conditions: SPINAL MUSCULAR ATROPHY TYPE 2
Phase 3 • Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated Sep 03, 2026 00:00 UTC