Can a second treatment boost motor skills in SMA kids after gene therapy?
NCT ID NCT04488133
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study tested the drug nusinersen (Spinraza) in 46 children under 3 years old with spinal muscular atrophy (SMA) who had previously received gene therapy but still had health challenges. The goal was to see if adding nusinersen could improve their muscle and movement abilities. Participants received multiple doses via lumbar puncture over nearly 2 years, and researchers measured motor milestones and safety.
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)
What this could lead to
If it works, this could show that adding nusinersen after gene therapy helps improve motor skills in young children with SMA.
What could go wrong
This is a small, completed phase 4 study with no control group, so results may not prove effectiveness. The treatment requires repeated lumbar punctures, which carry risks like headache or infection.
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, United States
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Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, 72202, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital Philadelphia - Neurology
Philadelphia, Pennsylvania, 19104, United States
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Children's Hospital of The King's Daughters
Norfolk, Virginia, 23510, United States
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Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Milan, 20133, Italy
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 00168, Italy
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Hospital Sant Joan de Déu
Esplugues Del Llobregat, Barcelona, 08950, Spain
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Hospital Universitario La paz
Madrid, 28046, Spain
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Oregon Health and Science University (OHSU)
Portland, Oregon, 97239, United States
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Schneider Children's Medical Center
Petah Tikva, 4920235, Israel
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Stanford Neuromuscular Research
Palo Alto, California, 94304, United States
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Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246, Germany
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University of Utah
Salt Lake City, Utah, 84112, United States