Gene therapy for newly diagnosed spinal muscular atrophy type I infants
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study tests SKG0201, a new gene therapy designed to treat spinal muscular atrophy type I (SMA type I), a rare genetic condition affecting muscle strength. The trial enrolls very young infants diagnosed with SMA type I to see if the treatment is safe and helps improve muscle function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your baby must have SMA type I, confirmed by genetic testing showing two copies of the mutated SMN1 gene
- Your baby must be 6 months old or younger on the day of treatment
- Your baby's oxygen level must be at least 96% when awake or sleeping without extra oxygen support
- Your baby's weight must be at the 3rd percentile or higher for their age and sex
- Your baby cannot have had another SMA gene therapy (like Zolgensma) or be in another SMA drug study
- Your baby cannot have active infections or need treatment for severe infections at the time of enrollment
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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