May 14, 2019

PPMD Hosts Duchenne Clinical Outcomes Workshop

PPMD has been working for many years to engage critical policy makers and stakeholders within the regulatory and access environments.  While we have learned a great deal over the past two years as we have…

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May 14, 2019

PPMD Awards $100,000 Grant to UCLA to Map the Immune Phenotype in Duchenne Muscular Dystrophy

PPMD is excited to announce that Dr. Carrie Miceli from the Center for Duchenne Muscular Dystrophy at University of California – Los Angeles (UCLA) is the recipient of a $100,000 grant to continue her work to…

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May 14, 2019

Solid Biosciences Provides Update on IGNITE DMD Status and New Clinical Sites

Solid Biosciences shared an update on the progress of their IGNITE DMD Phase I/II clinical trial for investigational microdystrophin gene transfer, SGT-001. The update includes news about initiating dosing for their higher dose cohort of…

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May 13, 2019

WATCH: Santhera Update to the Duchenne Community – Webinar Recording

On May 8, 2019, Santhera joined Parent Project Muscular Dystrophy for a webinar to provide the community with an update about respiratory health in Duchenne muscular dystrophy and the ongoing SIDEROS clinical trial. If you…

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May 9, 2019

Your Duchenne Registry Data: Researchers Analyze Steroid Use

We have exciting news to share from The Duchenne Registry. A study using the Registry data was published this week in a well-known international journal, BMC Neurology. This study was led by researchers at Catabasis…

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May 7, 2019

PPMD’s 2019 Annual Conference Agenda is Now Available!

PPMD is excited to unveil the agenda for our 2019 Annual Conference in Orlando, Florida, June 26 – June 30, 2019! Each year, PPMD works hard to put together a comprehensive agenda featuring updates in…

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May 3, 2019

End Duchenne Tour Recap: Portland, ME

PPMD had the opportunity to travel to Portland, ME on April 27 for our third End Duchenne Tour stop of 2019. We were joined by 14 local families, 11 of our industry partners, and 3…

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May 3, 2019

Newborn Screening Saves Lives Reauthorization Act Introduced in House of Representatives

Yesterday, the Newborn Screening Saves Lives Reauthorization Act was introduced in the House of Representatives. PPMD is extremely proud to be a leading coalition voice in this milestone legislation aimed at eliminating preventable newborn deaths…

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April 30, 2019

WATCH: Capricor’s Clinical Program – Webinar Recording

On Wednesday, April 24, 2019, Capricor joined Parent Project Muscular Dystrophy for a webinar to update the community on Capricor’s HOPE2 clinical program. If you missed the live event, the recording can be found below….

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April 26, 2019

PPMD Announces Univ. of Utah Health & Intermountain Primary Children’s as 25th Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named University of Utah Health (U of U Health) and the Intermountain Primary Children’s Hospital in Salt Lake City, Utah the 25th Certified Duchenne Care Center (CDCC). This expanding…

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