Gene therapy hope for babies with fatal muscle disease
NCT ID NCT06191354
First seen Nov 21, 2025 · Last updated Jun 02, 2026 · Updated 23 times
Summary
This study tests a new gene therapy called SKG0201 in 12 infants with spinal muscular atrophy type 1, a severe muscle-weakening disease. The treatment aims to improve survival and motor skills by delivering a working gene. Researchers are checking safety and how well the therapy works.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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National Children's Medical Center, Children's Hospital of Fudan University
Shanghai, China
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West China Sencond Hospital, Sichuan University / West China women's and children's Hospital
Chengdu, Sichuan, China
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Xinhua Hospital Affiliated To Shanghai Jiao Tong University School Of Medicine
Shanghai, 200092, China
Conditions
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