Gene therapy hope for babies with fatal muscle disease
NCT ID NCT07554924
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a gene therapy called SKG0201 in 11 infants with spinal muscular atrophy (SMA) type I, a severe muscle-weakening disease. The therapy uses a harmless virus to deliver a working copy of the missing SMN1 gene. The study aims to see if it is safe and can improve survival and motor skills.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
SKG0201 (gene therapy using a harmless virus to deliver a working SMN1 gene)
What this could lead to
If successful, this could provide a one-time treatment that improves motor function and survival in infants with SMA type I.
What could go wrong
This is an early, small trial (11 infants) testing safety and dosing. Gene therapy carries risks like immune reactions or liver issues, and it may not work for all.
Disclaimer
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the original study
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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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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.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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