One-Time gene therapy helps infants with devastating muscle disease sit and survive
NCT ID NCT03306277
Summary
This study tested a single-dose gene therapy for babies under 6 months old with a severe, life-threatening genetic muscle disease called Spinal Muscular Atrophy (SMA) Type 1. The goal was to see if the one-time intravenous (IV) treatment could help infants achieve major milestones like sitting independently and improve their survival without needing permanent breathing support. The therapy works by delivering a functional copy of a missing gene directly to the body's cells.
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 SMA - 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 and Robert H Lurie Children's Hospital
Chicago, Illinois, 60611, United States
-
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
-
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
-
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
-
Columbia University
New York, New York, 10032, United States
-
David Geffen School of Medicine at UCLA
Los Angeles, California, 90095, United States
-
Duke University
Durham, North Carolina, 27713, United States
-
Johns Hopkins Pediatric Neurology
Baltimore, Maryland, 21287, United States
-
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
-
Nemours Children's Hospital
Orlando, Florida, 32827, United States
-
Oregon Health and Science University
Portland, Oregon, 97239, United States
-
Stanford University
Stanford, California, 94305, United States
-
University of Texas Southwestern Medical Center
Dallas, Texas, 75235, United States
-
University of Utah
Salt Lake City, Utah, 84112, United States
-
University of Wisconsin (Madison)
Madison, Wisconsin, 53792, United States
-
Washington Unviersity School of Medicine
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.