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Exondys

This page shows the latest Exondys news and features for those working in and with pharma, biotech and healthcare.

FDA rejects Sarepta’s new Duchenne drug over safety fears

FDA rejects Sarepta’s new Duchenne drug over safety fears

Vyondys 53 (golodirsen) follows Exondys 51, the first and currently only disease-modifying treatment approved in the US for the inherited muscle-wasting disease. ... The question of clinical efficacy, which proved so controversial with Exondys has not

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