New gene therapy aims to help babies with deadly muscle disease
NCT ID NCT07554924
First seen May 02, 2026 · Last updated May 04, 2026 · Updated 1 time
Summary
This early-stage study tests a new medicine called SKG0201 in 11 babies with a severe muscle disease (spinal muscular atrophy type I). The goal is to see if it is safe and if it can improve muscle function. Babies must be under 6 months old to join.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
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Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
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Peking University First Hospital
Beijing, Beijing Municipality, China
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Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
Conditions
Explore the condition pages connected to this study.