New hope for SMA babies: add-on drug aims to boost motor skills after gene therapy
NCT ID NCT05861986
First seen Nov 01, 2025 · Last updated Apr 23, 2026 · Updated 22 times
Summary
This study tests whether adding risdiplam early can improve movement in children under 2 with spinal muscular atrophy who have already had gene therapy. About 28 children will receive risdiplam and be followed for 72 weeks. The goal is to see if the combination helps them reach motor milestones better than gene therapy alone.
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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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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
Conditions
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