Spinal injection drug shows promise in stopping SMA before symptoms start
NCT ID NCT02386553
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested a drug called nusinersen (Spinraza) in 25 infants who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The drug is given through a spinal injection to help delay or prevent the need for breathing support or early death. The goal was to see if early treatment can keep these babies healthier longer.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Nusinersen (Spinraza), a drug given via spinal injection
What this could lead to
If it works, this could help infants with SMA grow up without needing breathing support or dying early.
What could go wrong
This is a small, early-phase study with only 25 infants, so results may not apply to all. The drug requires repeated spinal injections, which carry risks like infection or headache.
Disclaimer
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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.
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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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-2605, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Columbia University
New York, New York, 10032, United States
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David Geffen School of Medicine
Los Angeles, California, 90095, United States
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Fondazione Serena Onlus - Centro Clinico Nemo
Milan, 20162, Italy
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Hacettepe University Medical Faculty
Ankara, 6230, Turkey (Türkiye)
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Hamad General Hospital
Doha, 3050, Qatar
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Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807, Taiwan
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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National Taiwan University Hospital
Taipei, 100, Taiwan
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Nemours Children's Hospital, Orlando
Orlando, Florida, 32827, United States
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Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 165, Italy
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Queensland Children's Hospital
South Brisbane, Queensland, 4101, Australia
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Royal Children's Hospital
Parkville, Victoria, 3052, Australia
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Seattle Children's Research Institute
Seattle, Washington, 98101, United States
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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The Johns Hopkins Hospital
Baltimore, Maryland, 21205, United States
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Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
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University of California Davis Health System
Sacramento, California, 95817, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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Yeditepe University Medical School Hospital
Istanbul, 31755, Turkey (Türkiye)