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3 Articles
3 Articles
GC301 gene therapy improves motor function in IOPD children
An experimental gene therapy known as GC301 improved motor function and cardiac outcomes for three out of four infants with infantile-onset Pompe disease (IOPD) in a small clinical trial. The treatment was also well tolerated. One year after treatment, these three participants had reached milestones such as walking with assistance. A fourth infant also received GC301, a treatment developed by Genecradle Therapeutics, but was withdrawn from the t…
Beijing – Chinese physicians have clinically tested gene therapy for the infantile variant of Pompe disease for the first time. According to their report in the New England Journal of Medicine (2025; DOI: 10.1056/NEJMoa2407766), three out of four infants achieve important milestones in motor development in their first year of life. Pompe disease is caused by...
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