New drug could help babies with SMA walk and sit normally
NCT ID NCT07221669
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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 helps the body make more of a protein that is missing in SMA, which is needed for muscles to work. Researchers want to see if starting treatment early can help babies reach normal milestones like sitting and walking, and prevent or reduce symptoms.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
salanersen (BIIB115)
What this could lead to
If successful, this could allow infants with SMA to reach normal motor milestones like sitting and walking, potentially preventing severe disability.
What could go wrong
This is an early-stage, small trial (30 infants) with no placebo group, so results may not be definitive. Long-term safety and efficacy are still unknown.
Disclaimer
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This is a summary of
the original study
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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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Ann and Robert H Lurie Childrens Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-••••
Contact
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Childrens Hospital of Fudan University_Shanghai
RECRUITINGShanghai, 201122, China
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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