Long-Term drug safety check for duchenne MD patients

NCT ID NCT01247207

Completed Disease control Sponsor: PTC Therapeutics Source: ClinicalTrials.gov ↗

First seen Mar 19, 2026 · Last updated May 08, 2026 · Updated 9 times

Summary

This study looked at the safety of a drug called ataluren in 270 people with Duchenne muscular dystrophy caused by a specific genetic mistake (nonsense mutation). Participants had already taken ataluren in earlier studies or were siblings of those who did. The goal was to see how safe the drug is over time, not to cure the disease. People stayed on the drug until it became available commercially or they chose to stop.

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Contacts and locations

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Locations

Alberta Children's Hospital

Calgary, Alberta, Canada
British Columbia Children's Hospital

Vancouver, British Columbia, Canada
CHU de Québec - Université Laval

Québec, Canada
Child Neurology Center of NW Florida NW

Gulf Breeze, Florida, 32561, United States
Children's Hospital Colorado - Center for Cancer and Blood Disorders

Aurora, Colorado, 80045, United States
Children's Hospital of Boston/Harvard Medical School

Boston, Massachusetts, 02115, United States
Children's Hospital of East Ontario

Ottawa, Ontario, Canada
Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of the King's Daughters

Norfolk, Virginia, 23507, United States
Childrens Medical Center Dallas

Dallas, Texas, 75207, United States
Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Columbia University Pediatric Neuromuscular Center

New York, New York, 10032, United States
Duke University Medical Center

Durham, North Carolina, 27710, United States
London Health Sciences Centre

London, Ontario, Canada
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States
Rare Disease Research, LLC

Atlanta, Georgia, 30329, United States
Rush Univ Medical Center

Chicago, Illinois, 60612, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Shriners Hospital for Children-Portland

Portland, Oregon, 97239, United States
Stanford University

Palo Alto, California, 94304, United States
Texas Children's Hospital

Houston, Texas, 77030, United States
UCLA

Los Angeles, California, 90095, United States
University of CA, San Francisco-Benioff Children's Hospital

San Francisco, California, 94158, United States
University of California-Davis

Sacramento, California, 95817, United States
University of Iowa Children's Hospital

Iowa City, Iowa, 52242, United States
University of Kansas Medical Center

Kansas City, Kansas, 66160, United States
University of Michigan

Ann Arbor, Michigan, 48109, United States
University of Minnesota

Minneapolis, Minnesota, 55455, United States
University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 16148, United States
University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States
University of Utah

Salt Lake City, Utah, 84112, United States
Washington University Medical School

St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.

DUCHENNE MUSCULAR DYSTROPHY