Children's Hospital is part of the UPMC family.
Be safe anytime, anywhere.
To find a pediatrician or pediatric specialist, please call 412-692-7337 or search our directory.
A resource for our network of referring physicians.
For more information about research, please call our main office at 412-692-6438.
Children's Hospital is ranked One of America's Best Children's Hospitals.
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
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
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
With myCHP, you can request appointments, review test results, and more.
For questions about a hospital bill call:
To pay your bill online, please visit UPMC's online bill payment system.
Interested in giving to Children's Hospital? Visit Children's Hospital of Pittsburgh Foundation's website to make a donation online.