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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:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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