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), ...
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 causes degeneration of skeletal and cardiac muscle ...
These findings were validated in mdx mice, a well-established animal model of DMD ... that aim to replace or repair the missing dystrophin protein, K884 focuses on bolstering the natural repair ...
Solid Bio's drug works by helping a patient's body make a miniature version of the dystrophin protein. Dystrophin keeps ... in several areas of the mouse model, we believe expression data should ...