Race to stop SMA before it starts: new drug tested in babies
NCT ID NCT07221669
Summary
This study is testing a drug called salanersen (BIIB115) in babies who have the genetic cause of spinal muscular atrophy (SMA) but have not yet shown any symptoms. The goal is to see if starting treatment very early can prevent or lessen the muscle weakness and movement problems caused by SMA. All participants will receive the drug via injection into the spinal fluid over several years, and researchers will track their ability to reach key movement milestones like sitting and walking.
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Contacts and locations
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Study contacts
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Contact
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Contact
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Locations
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Childrens Hospital of the Kings Daughter Norfolk
RECRUITINGNorfolk, Virginia, 23507, United States
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Contact
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Neurology Rare Disease Center
RECRUITINGFlower Mound, Texas, 75028, United States
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Conditions
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