Duchenne Muscular Dystrophy and MP-104

Expanded Access Protocol Intended to Provide Treatment With MP-104 (deflazacort) to U.S. Children, Adolescents, and/or Adults with Duchenne Muscular Dystrophy

Protocol Description

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.

Eligibility Criteria

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

Status: Open to Enrollment

Source(s) of Support

Marathon Pharmaceuticals

Primary Investigator

Hoda Abdel-Hamid, MD, MS

Contact Information

For more information about the study or enrollment, please contact:
Jennifer Monahan, Clinical Research Coordinator