Double treatment hope for SMA babies: gene therapy plus new drug
NCT ID NCT05861986
Summary
This study is testing if adding the drug risdiplam to babies who have already received gene therapy for spinal muscular atrophy (SMA) is safe and helps them improve their movement skills. It involves about 28 children under 2 years old. Researchers will measure changes in the babies' ability to sit, stand, and move over 72 weeks of treatment.
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.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Ann and Robert H. Lurie Children Hospital of Chicago
NOT_YET_RECRUITINGChicago, Illinois, 60611, United States
-
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
-
Columbia University Medical Center
RECRUITINGNew York, New York, 10032, United States
-
Cook Children's Jane and John Justin Neurosciences Center
RECRUITINGFort Worth, Texas, 76104, 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
-
The University of Texas Southwestern Medical Center at Dallas
RECRUITINGDallas, Texas, 75390, 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
-
Uniwersyteckie Centrum Kliniczne
RECRUITINGUniwersyteckie Centrum Kliniczne, 80-952, Poland
Conditions
Explore the condition pages connected to this study.