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New insights into protein roles in Duchenne muscular dystrophyinfluencing the stability of the dystrophin-associated protein complex across tissues, a key indication of DMD. These findings offer a molecular explanation for the wide-ranging symptoms ...
and some of the associated biochemical pathways. DMD is an X-linked disorder characterized by progressive muscle weakness and wasting due to the absence of a protein called dystrophin, which in turn ...
and the dystrophin-associated glycoprotein complex, which includes α-dystroglycan, the sarcoglycan subcomplex (α, β, γ,δ,and ε sarcoglycan), syntrophins, and dystrobrevins. Mutations in a number of ...
Solid Biosciences (SLDB) announced initial data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy ...
Dystrophin and DGC are also lost in Duchenne muscular dystrophy. The loss of dystrophin and damage to the DGC appear to be key players in the development of both cancer wasting and muscular dystrophy.
To understand the organization of the utrophin complex at neuromuscular junctions ... this connection because other syntrophin-associated proteins (dystrophin and utrophin) were less stringently ...
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