Experimental drug ataluren tested for safety in duchenne MD patients
NCT ID NCT01247207
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.
Disclaimer
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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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Alberta Children's Hospital
Calgary, Alberta, Canada
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British Columbia Children's Hospital
Vancouver, British Columbia, Canada
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CHU de Québec - Université Laval
Québec, Canada
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Child Neurology Center of NW Florida NW
Gulf Breeze, Florida, 32561, United States
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Children's Hospital Colorado - Center for Cancer and Blood Disorders
Aurora, Colorado, 80045, United States
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Children's Hospital of Boston/Harvard Medical School
Boston, Massachusetts, 02115, United States
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Children's Hospital of East Ontario
Ottawa, Ontario, Canada
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Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507, United States
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Childrens Medical Center Dallas
Dallas, Texas, 75207, United States
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Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Columbia University Pediatric Neuromuscular Center
New York, New York, 10032, United States
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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London Health Sciences Centre
London, Ontario, Canada
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Phoenix Childrens Hospital
Phoenix, Arizona, 85016, United States
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Rare Disease Research, LLC
Atlanta, Georgia, 30329, United States
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Rush Univ Medical Center
Chicago, Illinois, 60612, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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Shriners Hospital for Children-Portland
Portland, Oregon, 97239, United States
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Stanford University
Palo Alto, California, 94304, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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UCLA
Los Angeles, California, 90095, United States
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University of CA, San Francisco-Benioff Children's Hospital
San Francisco, California, 94158, United States
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University of California-Davis
Sacramento, California, 95817, United States
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University of Iowa Children's Hospital
Iowa City, Iowa, 52242, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
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University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 16148, United States
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University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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Washington University Medical School
St Louis, Missouri, 63110, United States