New Study Highlights Opportunities to Strengthen DM Clinical Trial Readiness
Clinical trial readiness is increasingly important as more potential myotonic dystrophy treatments advance through development. A new study accepted for publication in the Orphanet Journal of Rare Diseases explores what is needed to better prepare the DM community and research field for future trials and treatments.
Led by Third Plateau, “Exploring Barriers to Clinical Trial Readiness Among the Myotonic Dystrophy Community: A Mixed-Methods Study” brought together perspectives from people living with myotonic dystrophy, caregivers, pharmaceutical partners, principal investigators, and clinical research coordinators. Together, participants identified important barriers to clinical trial readiness and practical solutions that can help move the field forward.
Clinical Trial Readiness: Three Priorities for Action
The findings highlight three areas where coordinated action can make a meaningful difference:
- Developing clinical trial endpoints that better reflect the full lived experience of DM.
- Improving data sharing, registries, and coordination across the field.
- Reducing the logistical, financial, and accessibility burdens that can make trial participation difficult for individuals and families.
Importantly, the study reinforces a hopeful message: While the barriers facing the DM field are significant, they are also addressable.
Participants emphasized the need for more patient-centered outcome measures that capture the symptoms and challenges that matter in daily life, including fatigue, swallowing difficulties, cognitive changes, gastrointestinal symptoms, and overall function. The study also highlights the importance of stronger registries, better use of natural history and genetic data, improved trial site preparedness, and more inclusive trial designs that reduce burdens for participants and caregivers.
Preparing for the Next Stage of Therapeutic Development
The study comes at an important time for the myotonic dystrophy community. With multiple therapeutic programs advancing, the field has a unique opportunity to build the infrastructure needed for successful trials and, ultimately, future treatment access.
This work includes preparing clinical sites, improving recruitment pathways, supporting biomarker-driven studies, and ensuring that people living with DM and their caregivers remain central to research priorities.
MDF is proud to help convene these conversations and support a more coordinated, patient-centered approach to therapy development in DM. The study provides a roadmap for action across the entire field, including people living with DM, caregivers, clinicians, researchers, industry partners, regulators, and advocacy organizations. MDF has already committed to addressing several of these priorities through its new multiyear strategic plan, which will launch at the end of 2026.
Community Voices Are Essential
Most importantly, this work reflects the power of community input. People living with DM and their families are the experts in what meaningful improvement looks like. Their voices are essential to shaping better trials, better endpoints, and better pathways toward future therapies.
We are deeply grateful to everyone who contributed their time, experiences, and insights to this study. Together, we are helping prepare the DM community for the next stage of therapeutic development and building a stronger foundation for future progress.
Click here to read Exploring Barriers to Clinical Trial Readiness Among the Myotonic Dystrophy Community: A Mixed-Methods Study”.
