Molecular therapies in muscular dystrophies

Muscular dystrophies (MD) are a clinically and genetically heterogeneous group of skeletal muscle-wasting diseases with progressive muscle weakness and atrophy, while disease severity depends on the subtype of the disease. Tremendous progress in basic research and an improved understanding of the pathophyisology of the disease have led to various molecular pipeline therapies for MD. Within the last years, promising new molecular therapies have been developed facilitating causative therapy in the near future. New developments of personalized gene therapy aim at genetically defined disease subgroups of MD, based on the underlying molecular mechanism and the resulting phenotype, and set an example for other hereditary diseases. We have learned tremendously within the last decade; however, there is still a long way to go until these therapeutic strategies will be able to finally cure MD, and not just modify the phenotype and pathology of DMD patients.