Experimental drug ataluren tested for safety in duchenne MD patients

NCT ID NCT01247207

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested the safety of a drug called ataluren (also known as Translarna) in 270 people with Duchenne muscular dystrophy caused by a specific type of genetic mistake (nonsense mutation). Participants had already taken ataluren in earlier studies. The goal was to monitor side effects, lab results, and physical changes over time. The drug is meant to help the body produce a working version of the dystrophin protein, which is missing in Duchenne.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: ataluren (PTC124, Translarna)
What this could lead to: If successful, this could confirm that ataluren is safe enough to use long-term for managing Duchenne muscular dystrophy in people with a specific genetic mutation.
What could go wrong: This is an open-label safety study, not designed to prove the drug works. It only includes people who already took ataluren, so results may not apply to new patients. Side effects are still possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy neuromuscular disease caused by qualitative or quantitative defects of dystrophin

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY

Contacts and locations

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

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