Progress in the Treatment of Muscular Dystrophies

With a small patient population and a challenging breadth of disease types and underlying biological and genetic causes, the dystrophies have historically lacked attention from the biopharma industry as a value-driver for new drug development. New technical capabilities in gene therapy & gene editing and seed funds from philanthropic organizations focused on de-risking early stage drug development have changed the playing field.

In this report, we provide an overview of the current state of funding, therapeutics in development, and novel technical approaches towards treating this group of challenging diseases.

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