New gene therapy aims to help babies with deadly muscle disease
NCT ID NCT07554924
First seen May 02, 2026 · Last updated Jun 16, 2026 · Updated 10 times
Summary
This early-stage study tests a new medicine called SKG0201 in 11 babies with spinal muscular atrophy type I, a severe genetic condition that causes muscle weakness. The main goals are to check if the treatment is safe and to see if it can help babies survive and develop motor skills. The study is not yet open for enrollment.
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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
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.