Race to stop SMA before it starts: new drug trial for babies
NCT ID NCT07221669
Summary
This study is testing a drug called salanersen (BIIB115) in babies who have a genetic diagnosis of spinal muscular atrophy (SMA) but have not yet shown any symptoms. The goal is to see if starting treatment very early can prevent the onset of SMA symptoms or make them much less severe. Researchers will give the drug via spinal injection and closely monitor the babies' motor skill development, like sitting and walking, over several years.
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Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Childrens Hospital of the Kings Daughter Norfolk
RECRUITINGNorfolk, Virginia, 23507, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Email: •••••@•••••
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Neurology Rare Disease Center
RECRUITINGFlower Mound, Texas, 75028, United States
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Conditions
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