Can a Gene-Targeting drug stop SMA before it starts?
NCT ID NCT07221669
First seen Oct 31, 2025 · Last updated May 12, 2026 · Updated 26 times
Summary
This study tests a drug called salanersen in babies who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The drug aims to help the body make more of a protein needed for muscle and nerve function. Researchers want to see if early treatment can help babies reach normal motor milestones like sitting and walking.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
Contact Phone: •••-•••-••••
Contact
Conditions
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