Sarepta heading toward pivotal trial of first limb-girdle muscular dystrophy gene therapy

A new readout clears the path for Sarepta to move ahead next year with a pivotal study of its gene therapy for limb-girdle muscular dystrophy type 2E, one of several LGMD subtypes the company is seeking to treat.

The data also indirectly add to the body of evidence for the safety of

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