PTC Therapeutics Announces Publication of Data Demonstrating the Clinical Differentiated Benefit of Deflazacort | DuchenneXchange

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PTC Therapeutics Announces Publication of Data Demonstrating the Clinical Differentiated Benefit of Deflazacort


PTC Therapeutics, Inc. today announced the publication of data in Muscle & Nerve comparing the efficacy and safety of deflazacort and prednisone/prednisolone from the placebo arm of the ACT DMD study. The results demonstrated a clinically differentiated benefit of deflazacort over prednisone/prednisolone in slowing disease progression as measured using physical function endpoints and the time to delay loss of ambulation. Duchenne muscular dystrophy patients treated with deflazacort had notably less decline from baseline in 6-minute walk distance at Week 48 than those treated with prednisone/prednisolone. The extrapolated time to loss of ambulation was 8.58 years for deflazacort and 4.74 years with prednisone/prednisolone.

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