November 3, 2021

Record-Breaking 16th Annual Sam’s Night Raises Over $760,000 for PPMD

After the Killian family set the bar high in 2020 with over $532,000 raised at their annual Sam’s Night event, they managed to top that at this year’s event.  The 16th annual Sam’s Night has…

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November 3, 2021

Race Athlete Spotlight: Ernie Bueno

This November, Ernie Bueno will be running the NYC Marathon for the third year as part of the Race to End Duchenne team. He does it all for his friend and inspiration, Sam. Meet Ernie…

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November 2, 2021

Pfizer’s Phase 3 Gene Therapy Trial, CIFFREO: Community Questions Answered

Gene Therapy is on everyone’s mind these days. We wonder about opportunities, clinical sites, neutralizing antibodies and most recently details of a small number of Serious Adverse Events (SAE) and what these learnings might mean…

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November 1, 2021

November is National Family Caregivers Month

To be fair, I find it a little odd that we celebrate caregivers for only a single month, when caregivers are caring for their loved ones every single day of every single month. In this…

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November 1, 2021

Calling All Adults with Duchenne & Becker: Join the 2022 PPMD Adult Advisory Committee!

Been looking for a way to engage with the community a bit more? Have a personal experience that you would like to share with the larger community or federal and state advocacy efforts? Interested in…

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October 28, 2021

Edgewise Therapeutics shares positive topline results from EDG-5506 Phase 1 Study and doses first Becker patients

Edgewise Therapeutics, Inc. announced today positive topline results from the Multiple Ascending Dose (MAD) portion of a first-in-human Phase 1 clinical trial assessing the safety and tolerability of escalating doses of EDG-5506, an orally administered…

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October 27, 2021

Calling All Individuals with Becker Muscular Dystrophy – We Need You!

Do you or a loved one have a diagnosis of Becker muscular dystrophy? If so, please read these important updates which are focused on how we can help individuals with Becker and their families. Becker…

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October 22, 2021

Race Athlete Spotlight: Matthew Colon

Meet Matthew Colon Why did you choose to join the Race to End Duchenne team? Parent Project Muscular Dystrophy has given me hope for my sons future and for that, I am honored to be…

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October 18, 2021

WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update (Webinar Recording)

Last week, Parent Project Muscular Dystrophy was joined by Sarepta Therapeutics for a webinar to discuss the details of the EMBARK clinical trial (SRP-9001-301). If you missed the live event, the recording can be found below.*…

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October 14, 2021

The Duchenne Registry has a new survey – Therapy Services!

We know that therapy services such as physical, occupational, and speech therapy are helpful to people with Duchenne and Becker throughout their lives.   The new Therapy Services survey was developed to better understand who is…

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