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Sarepta’s Gene Therapy Impresses In Limb-Girdle Muscular Dystrophy

Pivotal Study Planned For 2021

 
• By John Davis

Positive functional results from Sarepta’s SRP-9003 gene therapy in limb-girdle muscular dystrophy type 2E bodes well for a pivotal study, and for the company’s studies of other potential gene therapies in Duchenne muscular dystrophy.

Skeletal_Muscle
Muscle tissue is affected in limb-girdle muscular dystrophy • Source: Shutterstock

Sarepta Therapeutics Inc. expects a pivotal study of its gene therapy, SRP-9003, for a specific type of limb-girdle muscular dystrophy (LGMD) to start in 2021, with the dose to be used decided upon by the third quarter of this year, following positive results from initial studies in six patients.

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