Muscular Dystrophy is a disabling condition, affecting primarily boys, that causes a mutation of the protein, dystrophin. This aberrant mutation of dystrophin, which acts like a molecular “shock absorber” for our muscles, in turn, leads to a host of debilitating outcomes: heart complications, difficulty walking, and trouble breathing.
Until now, little to no advance has been made to address the root cause of this disease.
Listen as Eventide CIO Finny Kuruvilla, MD, PhD, talks about exciting new research being done to tackle the root cause of muscular dystrophy, how the symptoms from this disease are being reversed, and why we believe Pediatric and Orphan Diseases to be a compelling investment theme.
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