Gene therapy hope for babies with fatal muscle disease
NCT ID NCT06191354
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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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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.
Contacts and locations
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
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Other studies related to the condition(s) this trial covers.