One-Shot gene therapy trial offers hope for infants with devastating muscle disease
NCT ID NCT07070999
Summary
This study is testing a single dose of an experimental gene therapy called GB221 for babies with spinal muscular atrophy type 1 (SMA1). The goal is to see if delivering a working copy of the missing SMN1 gene directly to the nervous system is safe and can help improve muscle function. It includes babies from 2 weeks to under 12 months old who already have symptoms, as well as younger babies who are at risk but don't yet show symptoms.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Hospital de Clínicas de Porto Alegre
RECRUITINGPorto Alegre, Rio Grande do Sul, 90035-903, Brazil
Conditions
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