Blog

PPMD: Your Strength, Your Family

PPMD: Your Strength, Your Family I had no intention of being part of the Duchenne community this long. When we started PPMD in 1994, I naively thought we would have a cure and I would…

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PPMD Awards $175,000 Grant to UCLA to Explore Optimization of CRISPR Technology in Duchenne

Parent Project Muscular Dystrophy (PPMD) is excited to announce that Drs. Melissa Spencer and April Pyle from the David Geffen School of Medicine at UCLA and the Center for Duchenne Muscular Dystrophy at University of…

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Jerry Mendell, MD named inaugural recipient of the Allen Distinguished Scholar in Pediatric Research

Nationwide Children’s Hospital has announced the inaugural recipient of the Allen Distinguished Scholar in Pediatric Research, Jerry Mendell, MD. Congratulations to Dr. Mendell on this incredibly deserved recognition! Thank you for your ongoing work in…

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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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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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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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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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PPMD Awards $100,000 Grant to Nationwide Children’s Hospital to Further Explore GALGT2 Gene Therapy

PPMD is excited to announce that Drs. Paul Martin and Kevin Flanigan from the Research Institute at Nationwide Children’s Hospital have completed work under a $100,000 grant exploring GALGT2 gene therapy technology as a potential treatment…

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Cure SMA & PPMD Announce Strategic Collaboration with Prometheus Research

PPMD is thrilled to announce our strategic collaboration with Cure SMA, one of the world’s leading rare disease patient advocacy organizations. Like PPMD, Cure SMA is looking to advance data collection and analysis on their…

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Updates from PPMD’s 2019 Gene Therapy Policy Forum

During PPMD’s Annual Advocacy Conference we had the opportunity to host a Gene Therapy Forum with expert clinicians, researchers, industry stakeholders, and government representatives taking part in the discussion. This meeting continues PPMD’s in-depth look…

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