New hope for SMA kids: extra drug boosts movement after gene therapy
NCT ID NCT04488133
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 22 times
Summary
This study looked at whether adding a medicine called nusinersen can help children under 3 with spinal muscular atrophy (SMA) who still have muscle problems even after receiving gene therapy. The 46 children received several doses of nusinersen over about 2 years. Researchers checked their motor skills (like sitting, crawling, and walking) and monitored for side effects. The goal was to see if the extra treatment could improve movement and safety.
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.
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
Locations
-
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
-
Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, 72202, United States
-
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
-
Children's Hospital Philadelphia - Neurology
Philadelphia, Pennsylvania, 19104, United States
-
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23510, United States
-
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Milan, 20133, Italy
-
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 00168, Italy
-
Hospital Sant Joan de Déu
Esplugues Del Llobregat, Barcelona, 08950, Spain
-
Hospital Universitario La paz
Madrid, 28046, Spain
-
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
-
Oregon Health and Science University (OHSU)
Portland, Oregon, 97239, United States
-
Schneider Children's Medical Center
Petah Tikva, 4920235, Israel
-
Stanford Neuromuscular Research
Palo Alto, California, 94304, United States
-
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246, Germany
-
University of Utah
Salt Lake City, Utah, 84112, United States
Conditions
Explore the condition pages connected to this study.