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The main objective of this multicenter study is to evaluate long-term safety and effects of Ataluren (PTC124) in treating muscle function in boys who have a certain type of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene. This study is an extension of a prior study and is only open to participants who completed that study.
Subject to certain exclusion criteria, the study is accepting male participants ages 7 to 18, who have a confirmed diagnosis of DMD caused by a nonsense mutation in the dystrophin gene, which causes a disruption in the body’s production of the dystrophin protein in muscle.
Boys: Ages 7 to 18
Participants will take ataluren 3 times every day in an amount appropriate for the individual’s body weight for up to 144 weeks (approximately 3 years). Other physical examinations, including physical assessments as well as biological sample collection, will be done at prescribed intervals. A final visit will be scheduled 6 weeks after the end of the dosing period.
Duration: About 3 years
Study Description at National Institutes of Health
Duchenne Muscular Dystrophy
Muscular Dystrophy Program at Children’s Hospital of Pittsburgh of UPMC
Hoda Abdel-Hamid, MD, MS
For more information about the study or enrollment, please contact:
Jennifer Monahan, Clinical Research Coordinator
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
Children’s Hospital of Pittsburgh of UPMC
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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