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Dyne Therapeutics

13,890 followers

1mo

Dyne partnered with Duchenne muscular dystrophy (#DMD) and myotonic dystrophy type 1 (#DM1) advocates across the globe to inform the design of our clinical trials and to reduce the burdens and inconveniences to trial participants and their families. These findings were published in the journal Research Involvement and Engagement. In honor of #WDAD24 and International #MyotonicDystrophy Awareness Day this month, we're excited to share an insightful conversation from Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy (PPMD) and Ashish Dugar, PhD, MBA, our Chief Medical Affairs Officer, about this collaboration. Watch the full video: https://bit.ly/3N3brVP and read the paper: https://bit.ly/4dMU8mW #clinicialtrials #patientcenteredresearch

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Transcript

- I'm super excited to be partnering with you to talk about the manuscript that we recently published together in the journal, Research, Involvement and Engagement. The manuscript is titled Patient Engagement and Clinical Trial Design for Rare Neuromuscular Disorders; impact on the DELIVER and ACHIEVE clinical trials, where you're the first author and Molly White and I who are with Dyne, are also authors. So what did you think when we first approached you with this idea to highlight this type of work that Dyne has done, and hopefully set some example or a bar for the industry. I'm just curious. What crossed your mind when we came to you? - When a family receives a diagnosis of a rare disease, you become by default advocates just because you have to inform your family, your community, your school, everyone who touches your life about that disease. So when you came to me and said, we want to do this manuscript because we want to really understand that patient voice, we're going to explore it with a patient community and really try to understand how we Dyne as a company can make things better in terms of our responsibilities and your burden. And how can we work together to figure out how to meet our needs, but also to understand what you're going through in order to make a decision to participate in a given trial. So, I see you taking all of that and creating an available model, something that people can sink their teeth in and go, ��Oh, that's how it works.��

SVP and Head of Clinical Development Operations

1mo

This is great work and something we should all be striving towards!

Tammy Burke, EMBA, BSN, RN

Tammy Burke, EMBA, BSN, RN

Sr. Director, Patient Recruitment, Feasibility & Engagement

1mo

Congratulations! Great work. So happy to follow this!

Mary-Jean Fanelli, MD, MBA

Mary-Jean Fanelli, MD, MBA

Strategic Medical Affairs Leader | Driving Medical Transformation | Building & Connecting Teams for Impact and Results | Collaborative Leadership

1mo

Fantastic work with the patient community. Very exciting!

Laura Genatossio

Laura Genatossio

Senior Vice President, General Manager Sumitomo Pharma Switzerland

1mo

Great conversation!

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