October 14, 2020

Publication of Australian Consensus Statement for Powered Wheelchair Standing Devices in Duchenne

As many families know, getting a Powered Wheelchair Standing Device (PWSD) approved and covered by insurance can be a frustrating and exhausting task. Having the same issues in Australia, an international multidisciplinary panel of clinicians,…

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October 12, 2020

Italfarmaco Receives FDA Rare Pediatric Disease Designation & Completes EPIDYS Phase 3 Trial Enrollment

Italfarmaco has provided an update on the development of Givinostat, sharing that the FDA has granted a Rare Pediatric Disease designation to Givinostat for the treatment of Duchenne, which allows an expedited review process for…

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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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