exondys 51

Sarepta Therapeutics Shares COVID-19 Resources

Sarepta Therapeutics has shared a new COVID-19 resource page on its website, along with a new video providing contact phone numbers for those whom may have questions about continuity of access to Sarepta sponsored clinical…

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Sarepta Therapeutics Shares Community Update on COVID-19

Sarepta Therapeutics has provided the community with an update addressing questions from the community regarding COVID-19, including updates on current clinical trials and accessing approved therapies. Read Sarepta’s Update March 19, 2020 Dear Duchenne and…

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August 16, 2019

ICER Releases Report; PPMD Issues Open Letter to Payers

Last night, the Institute for Clinical and Economic Review (ICER) released their final report assessing three Duchenne products: Emflaza, EXONDYS 51, and golodirsen. This report integrates all public comments, stakeholder engagement, and the Roundtable proceedings…

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February 19, 2019

End Duchenne Tour Recap: Las Vegas, NV

PPMD was thrilled to head to Las Vegas, NV for our first End Duchenne Tour stop of 2019 on February 2. More than 45 people came together to learn about the latest information and resources for Duchenne…

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Sarepta Shares Quarterly Update, Including US Clinical Trial Updates

Sarepta has provided another quarterly update to the community, including information on their PPMO (Peptide Conjugated Phosphorodiamidate Morpholino Oligomer), an exon skipping technology designed to efficiently get inside cells, with the goal of producing more dystrophin in…

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