Hope for SMA babies: new drug may restore movement after gene therapy falters
NCT ID NCT05861999
First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 29 times
Summary
This study tests a medicine called risdiplam in babies under 2 years old with spinal muscular atrophy (SMA) who stopped getting better or started losing skills after receiving gene therapy. The goal is to see if risdiplam can help them regain motor abilities like sitting and moving. About 28 children will take part, and researchers will track their progress over 72 weeks using a standard motor skills test.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for MUSCULAR ATROPHY, SPINAL are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
RECRUITINGBerlin, 13353, Germany
-
Children'S Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
-
Children's Healthcare of Atlanta Center for Advanced Pediatrics
RECRUITINGAtlanta, Georgia, 30329-2309, United States
-
Children's Hospital of Colorado
RECRUITINGAurora, Colorado, 80045, United States
-
Children's Hospital of the King's Daughter
RECRUITINGNorfolk, Virginia, 23510, United States
-
Great Ormond Street Hospital For Children
RECRUITINGLondon, WC1N 3JH, United Kingdom
-
Helen DeVos Children's Hospital at Spectrum Health
RECRUITINGGrand Rapids, Michigan, 49503, United States
-
Instytut Pomnik Centrum Zdrowia Dziecka
RECRUITINGWarsaw, 04-730, Poland
-
Schneider Children's Medical Center of Israel
RECRUITINGPetah Tikva, 4920235, Israel
-
Sidra Medicine
RECRUITINGAl Rayyan, Qatar
-
Soroka Medical Center
RECRUITINGBeersheba, 8410101, Israel
-
Sourasky MC, Dana-Dwek Children's Hospital
RECRUITINGTel Aviv, 6423906, Israel
-
Stanford Univ Medical Center
RECRUITINGPalo Alto, California, 94304, United States
-
UKGM Standort Gießen
RECRUITINGGiessen, 35392, Germany
-
University of Arkansas for Medical Sciences
RECRUITINGLittle Rock, Arkansas, 72103, United States
-
University of Florida Pediatrics
RECRUITINGGainesville, Florida, 32610, United States
-
University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
-
Uniwersyteckie Centrum Kliniczne
RECRUITINGGdansk, 80-952, Poland
-
Valley Children's Hospital
RECRUITINGMadera, California, 93636, United States
Conditions
Explore the condition pages connected to this study.