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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October 4, 2018

Jerry Mendell, M.D. Shares Positive Updated Results from Gene Therapy Micro-dystrophin Trial

  PPMD is excited to learn that Dr. Jerry Mendell of Nationwide Children’s Hospital today shared positive results for the fourth patient in Sarepta’s micro-dystrophin trial, indicating positive functional signals for all of the patients….

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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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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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December 19, 2017

Understanding Currently Recruiting Gene Therapy Studies

For the last many months, we have all been watching gene therapy make headlines around the world, across various diseases. As you know, earlier this year PPMD launched a Gene Therapy Initiative to help support the exploration…

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January 6, 2017

PPMD Awards Nationwide Children’s Hospital $2.2 Million Grant to Explore Gene Therapy in Duchenne

Today, PPMD announced a $2.2 million dollar grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital.  Our purpose in awarding this grant is to open and accelerate the field of gene…

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