New combo approach aims to boost motor skills in babies with SMA after gene therapy
NCT ID NCT05861986
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 26 times
Summary
This study tests whether adding risdiplam early can improve motor skills in infants under 2 with spinal muscular atrophy who already received gene therapy. About 28 children with two SMN2 gene copies will take risdiplam for 72 weeks. Researchers will track changes in gross motor scores and monitor side effects.
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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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