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This multicenter study provides MP-104, also known as deflazacort, a glucocorticosteroid, for people with Duchenne muscular dystrophy (DMD). Deflazacort is approved outside of the United States for use in anti-inflammatory and immunosuppressant indications, and is being investigated for treatment in patients with DMD.
Subject to certain exclusion criteria, the study is accepting participants of both genders, ages 5 through seniors, who have a confirmed diagnosis of DMD, whose physicians think they might benefit from taking deflazacourt for allergic and inflammatory conditions.
Males and Females: Age 5 and up
Participants in this study will receive either a tablet or oral solution of deflazacort, as determined by the treating physicians, who will also monitor patient responses to the medication.
Visits: None beyond normal care for DMD
Duration: Up to 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.
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