One-Shot gene therapy helps babies with fatal muscle disease sit up and survive
NCT ID NCT03306277
Summary
This study tested a single-dose gene therapy for babies under 6 months old with spinal muscular atrophy type 1, a severe genetic muscle-wasting disease. The goal was to see if the one-time intravenous treatment could help babies achieve major milestones like sitting independently and improve survival without permanent breathing support. The trial enrolled 22 infants to measure these key outcomes.
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 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