One-Shot gene therapy offers hope for kids with rare, devastating movement disorder
NCT ID NCT06692712
Summary
This study is testing a one-time gene therapy injection for young children with a rare, inherited condition called SPG50 that severely affects movement and development. The therapy aims to deliver a working copy of the faulty gene directly to the nervous system to potentially slow or stop disease progression. Researchers will compare 24 children who receive the treatment to a similar group who do not, measuring changes in motor skills like sitting, crawling, and walking over three years.
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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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Sant Joan de Deu
ACTIVE_NOT_RECRUITINGBarcelona, 08950, Spain
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University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75025, United States
Conditions
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