December 10, 2020

The Duchenne Registry Celebrates One Year of Data Collection in our Smartphone App!

This year has been one for the record books for many reasons, but here at PPMD we are excited to celebrate one year of data collection in The Duchenne Registry’s new smartphone app platform! Our…

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December 9, 2020

What exactly is a “Duchenne Protein Map”?

One thing we all know about Duchenne is that no matter when you or your child was diagnosed, some days we wake up and find ourselves feeling like there is still so much we need…

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December 7, 2020

Support PPMD to Unleash Breakthroughs

It is hard to believe that we are here — in the throes of the “magic” of the holiday season. 2020 has not felt much like magic. Instead, a roller coaster of unpredictability and worry. But…

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December 7, 2020

Sarepta Announces Positive Clinical Results from MOMENTUM Phase 2 Clinical Trial of SRP-5051

Sarepta has announced positive results from the ongoing MOMENTUM study of SRP-5051, its next-generation treatment for individuals with Duchenne who are amenable to exon 51 skipping. This is the first clinical data from SRP-5051, an…

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December 3, 2020

WATCH: SRP-9001 Gene Therapy Path Forward (Webinar Recording)

Parent Project Muscular Dystrophy and Sarepta Therapeutics hosted a webinar on November 24, 2020 for a discussion about the SRP-9001 gene therapy development program. If you missed the live event, the recording can be found…

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November 25, 2020

I’m thankful for what we have in each other.

This year. This Thanksgiving. How did we get here? This year has tested our will, our resilience, and our courage. I still have a turkey in the oven and dessert in the fridge, but my…

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November 25, 2020

Antisense Therapeutics: Introduction to the Duchenne Community

PPMD is thrilled to welcome Antisense Therapeutics to the Duchenne community. Antisense is developing a new potential therapy, ATL1102, using an antisense oligonucleotide (ASO) inhibitor aimed at treating muscle inflammation and associated muscle wasting. Although…

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November 22, 2020

Lilly and Precision BioSciences Announce Genome Editing Research Collaboration and License Agreement

PPMD is excited to welcome Lilly back to the Duchenne space! With Lilly’s experience in Duchenne, we are thrilled to see their return to the field and look forward to learning more as this early…

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November 21, 2020

NEW – Fall 2020 Survey: Impact of the COVID-19 Pandemic on Duchenne

PPMD has launched our second COVID-19 Pandemic Impact Survey related to your experiences during the pandemic for accessing care, trials, and approved therapies. Thank you to everyone who completed our first survey earlier in the spring….

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November 20, 2020

Attend PPMD’s Community Concert: Harmony Together

Join us on Sunday, November 22 at 7 PM EST for an incredible evening of music on the PPMD Facebook Page! PPMD’s Community Concert: Harmony Together will feature members of the Duchenne community sharing their…

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