A new study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein, dystrobrevin, offering new pathways for understanding and ...

Newer therapies like PGN-EDO51 that target exon skipping are considered promising pathways to slow disease progression by restoring partial dystrophin function.

Revidia Therapeutics Inc., a cardiac regenerative medicine company developing first-in-class small molecule drug therapies for heart injury, has announced that it has expanded its leadership team in ...

A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein, dystrobrevin, offering new pathways for ...

These include gene-based therapies (e.g., replacing a patient's faulty DMD genes with normally functioning ones), cell-based therapies (e.g., replacing dystrophin-deficient muscle cells with stem ...

At the moment the only approved therapies are exon-skipping or stop codon read-through drugs, designed to restore dystrophin function in DMD patients with specific mutations in the dystrophin gene.

Capricor's biologics license application for deramiocel (CAP-1002) is draws largely on the Phase 2 HOPE-2 trial and its ...

Studies involving the selective restoration of membrane bound nNOS to dystrophin-deficient skeletal muscle will address some of these issues. In addition to shedding light on the function of the ...