PTC Therapeutics 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.
https://bit.ly/2LkaDhG
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