Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the ...
Clinical Trials Arena on MSN18d
Dyne’s DMD trial advances patient mobility and dystrophin expressionThe Phase I/II Deliver trial sought to establish the impact of DYNE-251 on several mobility-related endpoints, seeing an increase over baseline at 12 months.
Dystrophin is part of a protein complex linking the cytoskeleton of muscle fibers to the surrounding connective tissue (basal lamina). It’s a long protein with numerous redundant coils (purple balls), ...
Precision Biosciences Inc. recently presented a new gene-editing approach, PBGENE-DMD, which could allow life-long benefits to patients with Duchenne muscular dystrophy (DMD).
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