One-Time gene infusion aims to save babies from devastating muscle disease
NCT ID NCT03837184
Summary
This study tested a single-dose gene replacement therapy for babies under 6 months old with a severe form of spinal muscular atrophy (SMA Type 1). The main goal was to see if the treatment could help these infants achieve the milestone of sitting up on their own for at least 10 seconds by 18 months of age. The therapy, delivered through a one-hour IV infusion, aims to address the genetic root cause of the disease.
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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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National Taiwan University Hospital
Taipei, Taiwan
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Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, South Korea
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Seoul National University Hospital
Seoul, South Korea
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Tokyo Women's Medical University
Tokyo, Japan
Conditions
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