October 9, 2020

Connect with the Experts: Endocrine Issues in Duchenne

The body’s endocrine system is made up of glands that produce hormones, which are chemical messengers. Examples are growth hormone (that tells your body when to start and stop growing) and testosterone (the male hormone…

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October 8, 2020

2020 World Muscle Society: Duchenne Research Updates

Last week the World Muscle Society (WMS) held its 25th Annual Conference. As is often the case during this pandemic, the WMS meeting was held virtually for the first time. WMS provides an opportunity for…

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October 8, 2020

Sarepta Therapeutics Shares Fall 2020 Newsletter

Sarepta has shared the Fall 2020 issue of their newsletter, providing updates on community programs and highlighting their new class of Route 79 scholarship recipients. Click here to download.

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October 7, 2020

End Duchenne Tour 2020: Virtual Tour Stop in Portland

End Duchenne Tour 2020: Virtual Tour Stop in Portland, OR On Saturday, September 12, we spent the morning with the Duchenne community in Portland, Oregon and the surrounding region for another End Duchenne [Remote] Tour…

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October 6, 2020

First Results from PPMD/Nationwide Carrier Study – Evidence of Cardiomyopathy by Exercise & Cardiac MRI

For females living with dystrophinopathy (the spectrum of diseases caused by mutations in the DMD gene), the disorder has not been historically characterized effectively. To address this need, PPMD provided a grant to Nationwide Children’s…

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October 6, 2020

Santhera to Discontinue Phase 3 SIDEROS Study and Development of Puldysa® in Duchenne

Today, we are disappointed to learn that Santhera announced its decision to discontinue development of its Phase 3 SIDEROS study with Puldysa® (idebenone) in Duchenne. Santhera will stop the SIDEROS trial (including extension) and participants…

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October 2, 2020

Catabasis Shares October 2020 Newsletter

Catabasis Pharmaceuticals has shared the latest edition of their community newsletter which includes information from their presentations at the Congress of the World Muscle Society. The Phase 3 PolarisDMD trial and GalaxyDMD open-label extension trial…

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October 1, 2020

Coach To Cure MD: Season 12 Is Underway!

It’s the start of the football season and we are excited to kick off this year’s 12th Annual Coach To Cure MD! Although we would normally host our annual event on the last Saturday of September,…

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October 1, 2020

Pfizer Receives FDA Fast Track Designation for PF-06939926 Investigational Gene Therapy

Pfizer Inc. announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne has received Fast Track designation from the FDA. PF-06939926 is currently being evaluated to determine the safety and efficacy of…

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October 1, 2020

FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial

PPMD is delighted to learn that the FDA has lifted the hold on Solid Biosciences’ IGNITE-DMD clinical trial for their gene therapy product (SGT-001) delivering a micro-dystrophin transgene via AAV9. Solid has worked diligently to…

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