New drug may boost motor skills in kids with SMA after gene therapy
NCT ID NCT05861986
First seen Nov 01, 2025 · Last updated Jun 20, 2026 · Updated 31 times
Summary
This study is testing whether the drug risdiplam can improve motor skills in children under 2 with spinal muscular atrophy (SMA) who have already received gene therapy. The 28 participants will take risdiplam by mouth, and researchers will measure changes in their gross motor skills over 72 weeks. The goal is to see if adding this medication after gene therapy provides extra benefit.
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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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann and Robert H. Lurie Children Hospital of Chicago
NOT_YET_RECRUITINGChicago, Illinois, 60611, United States
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Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
RECRUITINGBerlin, 13353, Germany
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Children'S Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Children's Healthcare of Atlanta Center for Advanced Pediatrics
RECRUITINGAtlanta, Georgia, 30329-2309, United States
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Children's Hospital of Colorado
RECRUITINGAurora, Colorado, 80045, United States
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Children's Hospital of the King's Daughter
RECRUITINGNorfolk, Virginia, 23510, United States
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Columbia University Medical Center
RECRUITINGNew York, New York, 10032, United States
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Cook Children's Jane and John Justin Neurosciences Center
RECRUITINGFort Worth, Texas, 76104, United States
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Great Ormond Street Hospital For Children
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Helen DeVos Children's Hospital at Spectrum Health
RECRUITINGGrand Rapids, Michigan, 49503, United States
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Instytut Pomnik Centrum Zdrowia Dziecka
RECRUITINGWarsaw, 04-730, Poland
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The University of Texas Southwestern Medical Center at Dallas
RECRUITINGDallas, Texas, 75390, United States
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UKGM Standort Gießen
RECRUITINGGiessen, 35392, Germany
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University of Arkansas for Medical Sciences
RECRUITINGLittle Rock, Arkansas, 72103, United States
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University of Florida Pediatrics
RECRUITINGGainesville, Florida, 32610, United States
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Uniwersyteckie Centrum Kliniczne
RECRUITINGUniwersyteckie Centrum Kliniczne, 80-952, Poland
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
risdiplam
What this could lead to
If it works, this could show that adding risdiplam after gene therapy helps young children with SMA gain better motor skills.
What could go wrong
This is a small, early-phase study with only 28 participants, so results may not apply to all children. Risks include side effects from the drug.
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.