Can a second treatment help kids with SMA who hit a plateau?
NCT ID NCT05861999
First seen Nov 01, 2025 · Last updated May 05, 2026 · Updated 28 times
Summary
This study is for children under 2 with spinal muscular atrophy (SMA) who received gene therapy but then stopped getting better or started to decline. Researchers want to see if adding a medicine called risdiplam can help them regain motor skills like sitting or crawling. The study will follow 28 children for 72 weeks to check safety and effectiveness.
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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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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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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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Schneider Children's Medical Center of Israel
RECRUITINGPetah Tikva, 4920235, Israel
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Sidra Medicine
RECRUITINGAl Rayyan, Qatar
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Soroka Medical Center
RECRUITINGBeersheba, 8410101, Israel
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Sourasky MC, Dana-Dwek Children's Hospital
RECRUITINGTel Aviv, 6423906, Israel
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Stanford Univ Medical Center
RECRUITINGPalo Alto, California, 94304, 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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University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
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Uniwersyteckie Centrum Kliniczne
RECRUITINGGdansk, 80-952, Poland
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Valley Children's Hospital
RECRUITINGMadera, California, 93636, United States
Conditions
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