Sarepta Therapeutics Inc.

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Genetic Modification Therapies Clinical Applications (BIO159B)

EXONDYS 51		EXONDYS 51 is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene. EXONDYS 51 is based on exon skipping technology, which skips exon 51 of the dystrophin gene.
VYONDYS 53		VYONDYS 53 was launched in 2019 for the treatment of Duchenne in patients who have a mutation of the dystrophin gene. VYONDYS 53 is based on the company’s PMO chemistry and uses exon-skipping technology to skip exon 53 of the dystrophin gene.
AMONDYS 45		AMONDYS 45, launched in 2021, is indicated for the treatment of Duchenne muscular dystrophy in patients. It is suitable for patients with a confirmed mutation of the dystrophin gene that skips exon 45.

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