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0:23
YouTube
NEJM Group
Brief Report: Infants’ Motor Development after Gene Therapy for Pompe’s Disease (Patient 1)
Before the injection of GC301 gene therapy, the 3-month-old patient had mouth breathing, was unable to lift the head, and had low limb-muscle tone. One month after the injection, the 4-month-old patient was able to lift the head to 90 degrees in the prone position and lift the chest. Seven months after the injection, the 10-month-old patient ...
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