Sarah, who is living with myotonic dystrophy type 1 (DM1), a disease with no approved therapies, and our Chief Medical Officer, Wildon Farwell, discuss how a meaningful therapeutic option could change her life and the challenges of treating DM1.
Our Focus:
Our Commitment
We support all people, families and caregivers living with rare diseases, this Rare Disease Day and every day
At Dyne, we are committed to listening to and learning from the communities we serve—including individuals and families living with DM1, DMD and FSHD. Your stories inspire us. Equally important, they provide insights and information about disease burden and meaningful benefit that we know is critical to developing truly transformative therapies.
Letters to Our Communities
Below are links to correspondence we’ve written to individuals and families affected by the diseases we focus on, addressing significant events and topics.
Community Insights Publication: Patient Engagement in Clinical Trial Design for Rare Neuromuscular Disorders
Joining Forces is a series of conversations between community members and Dynamos. Through these discussions, we aim to learn more about the impact their disease has on their daily lives, their hopes for the future, and to answer questions about Dyne’s efforts to advance potentially life-transforming therapies. Through our endeavors and collective experiences, we are stronger together.
Sarah and Wildon | Episode 1
Alan and Ash | Episode 2
Alan, who is living with Duchenne muscular dystrophy (DMD), and our Chief Medical Affairs Officer, Ash Dugar, discuss one of the biggest unmet needs of the DMD community and how critical the community voice and their perspectives are to Dyne.
Pat and Debra | Episode 3
Pat, who is an Advisor for Parent Project Muscular Dystrophy’s Adult. Advisory Committee and is living with muscular dystrophy and our Chief Regulatory Affairs Officer, Debra Feldman, discuss Regulatory’s role in developing therapeutics for rare muscle diseases and what Dynamos should know as we work on advancing life-transforming therapies for people living with genetically driven diseases.
Pat and Ash | Episode 4
President and Chief Executive Officer of Parent Project Muscular Dystrophy, Pat Furlong, and our Chief Medical Affairs Officer, Ash Dugar discuss their collaborative publication published in Research Involvement and Engagement on the importance of patient engagement in clinical trial design.
Each person living with DM1, DMD or FSHD has a unique and powerful story to tell
Rare Disease Day
Each February, Rare Disease Day aims to raise awareness of the impact that rare diseases have on over 300 million people around the world.
2024
Dyne hosted members of the rare muscle disease community, Dynamos and their families to commemorate Rare Disease Day. At Dyne, our community members are experts who continue to educate us about their disease and experience. Embracing the “All Access” theme, individuals from DMD, DM1, and FSHD communities led education booths, sharing valuable information and insights with our Dynamos.
2023
Members of the rare muscle disease community, Dynamos and their families joined together to mark Rare Disease Day at Dyne. Watch the video to hear their perspectives on the importance of recognizing Rare Disease Day and Dyne’s commitment to delivering for patients.
2022
2021
2020
Dyne and Notre Dame: A shared commitment to patient advocacy
Dyne has partnered with Notre Dame to support its minor in science and patient advocacy, a unique program to prepare the next generation of leaders for careers focused on improving patient care. Since September 2021 Dyne has collaborated with Notre Dame to convene a Rare Patient Advocacy Summit at the university, followed by hosting two students as summer interns.
2023
2022
2021
Watch students from the Class of 2022, Bailey and Alyssa, as well as leaders from Notre Dame and Dyne, describe the importance of incorporating community members’ voices in the drug development process.
