First human trial of 'Tianasen' offers hope for kids with devastating genetic disorder
NCT ID NCT07363603
Summary
This study is testing a new drug called Tianasen (ASO-GNAO1) for children with a severe genetic brain disorder caused by a specific GNAO1 gene mutation. The drug is designed to target and reduce the harmful protein produced by the faulty gene, with the goal of slowing or stopping the progression of seizures and movement problems. Five children, aged 1 to 14, will receive the drug via spinal injections over a year while doctors closely monitor their symptoms and safety.
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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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Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University
RECRUITINGMoscow, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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