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Sarepta’s Duchenne gene therapy misses main goal in late-stage study; shares fall

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(Reuters) -Sarepta said on Monday that its late-stage study testing two gene-targeted therapies for Duchenne muscular dystrophy did not meet the main goal, sending its shares plummeting 21% in extended trading.

The study tested casimersen and golodirsen in 225 boys aged 6 to 13 with a rare form of Duchenne that can be treated by skipping exons 45 or 53.

The trial ran for nine years and was affected by the COVID-19 pandemic, which the company said impacted results.

Sarepta said the therapies were well tolerated, with no new safety concerns.

(Reporting by Kamal Choudhury in Bengaluru; Editing by Alan Barona)

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