September 24, 2018

Sarepta Therapeutics Announces Clinical Hold Lifted for its Micro-dystrophin Gene Therapy Program

We are thrilled to learn that the FDA has lifted the clinical hold for Sarepta’s Duchenne micro-dystrophin gene therapy program. Sarepta previously announced on July 25, 2018, that the FDA placed the program on clinical…

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July 25, 2018

Sarepta Therapeutics Announces Clinical Hold on Micro-Dystrophin Gene Therapy Trial

This afternoon Sarepta Therapeutics, Inc. announced that the micro-dystrophin Phase 1/2a gene therapy trial was placed on clinical hold by the FDA as the result of an out-of-specification production lot. The company was notified by the Research…

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June 19, 2018

Positive Preliminary Results from the First Three Children Dosed in Phase 1/2a Gene Therapy Micro-dystrophin Trial

Sarepta Therapeutics announced that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from its Phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne. Ryan…

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June 8, 2018

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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May 31, 2018

WATCH: Introducing PPMO – Webinar Recording with Sarepta Therapeutics

Sarepta Therapeutics, Inc. recently joined Parent Project Muscular Dystrophy (PPMD) for a community webinar update to discuss PPMO, Sarepta’s next generation precision RNA-targeting therapeutic platform for use in Duchenne muscular dystrophy. During the webinar, Sarepta…

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May 2, 2018

May 9 Webinar: Introducing PPMO – The Future of Precision RNA-Targeted Therapies for Duchenne

Sarepta Therapeutics, Inc. will join Parent Project Muscular Dystrophy for a webinar update on Wednesday, May 9 at 1:00 PM EDT to discuss PPMO, Sarepta’s next generation precision RNA-targeting therapeutic platform for use in Duchenne…

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January 10, 2018

First Duchenne Patient Dosed in Microdystrophin Gene Therapy

“DMD gene therapy went well. It was started at 1:15 PM and ended at 2:27 PM”     On Thursday, January 4, I received this message from Dr. Jerry Mendell accompanied by the photo above – Dr….

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