Early treatment may stall SMA: study shows promise in infants
NCT ID NCT02386553
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 33 times
Summary
This study tested a drug called nusinersen in 25 babies who have the genetic markers for spinal muscular atrophy (SMA) but no symptoms yet. The goal was to see if early treatment could delay or prevent the need for breathing help or death. The drug is given via a spinal injection, and the study followed infants for key milestones like survival and symptom onset.
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 SPINAL MUSCULAR ATROPHY 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
Locations
-
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-2605, United States
-
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
-
Columbia University
New York, New York, 10032, United States
-
David Geffen School of Medicine
Los Angeles, California, 90095, United States
-
Fondazione Serena Onlus - Centro Clinico Nemo
Milan, 20162, Italy
-
Hacettepe University Medical Faculty
Ankara, 6230, Turkey (Türkiye)
-
Hamad General Hospital
Doha, 3050, Qatar
-
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807, Taiwan
-
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
-
National Taiwan University Hospital
Taipei, 100, Taiwan
-
Nemours Children's Hospital, Orlando
Orlando, Florida, 32827, United States
-
Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 165, Italy
-
Queensland Children's Hospital
South Brisbane, Queensland, 4101, Australia
-
Royal Children's Hospital
Parkville, Victoria, 3052, Australia
-
Seattle Children's Research Institute
Seattle, Washington, 98101, United States
-
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
-
The Johns Hopkins Hospital
Baltimore, Maryland, 21205, United States
-
Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
-
University of California Davis Health System
Sacramento, California, 95817, United States
-
University of Utah
Salt Lake City, Utah, 84112, United States
-
Yeditepe University Medical School Hospital
Istanbul, 31755, Turkey (Türkiye)
Conditions
Explore the condition pages connected to this study.