Resources for Study & Trial Participants | Myotonic Dystrophy Foundation

Clinical Trials Training for Trial Participants
Communications Behavior for Trial Participants
An Ounce of Prevention: Social Media Discussions and Clinical Trial Threats
Clinical Trials 101: What They Are, How They Work, Dr. Bruce Wentworth, Sarepta; Jeanne Dekdebrun, Elizabeth Luebbe, University of Rochester
The US Drug Testing & Approval Process, Dr. Doug Kerr, Biogen Idec; related PowerPoint presentation
The Clinical Trial: Real Life Experiences, Pat Furlong, Parent Project Muscular Dystrophy; related PowerPoint presentation
How Antisense Oligoneucleotide Treatments are Designed to Work, Dr. Bruce Wentworth, Genzyme; related Powerpoint presentation
DM Clinical Trial Readiness, Dr. Charles Thornton, University of Rochester; related PowerPoint presentation

If you have additional questions about the clinical trial process, please contact MDF.

Myotonic Dystrophy News

April 2,2024

Participants Needed for Important DM Research Study

The Myotonic Dystrophy Clinical Research Network (DMCRN) sites are currently conducting a critically-important research study designed to help drug developers successfully design clinical trials and understand how to assess the efficacy of potential therapies.

March 29,2024

Request for Applications Open - 2025 MDF Research Fellowships & Grants!

We are thrilled to announce the upcoming launch in April of four grant programs aimed at accelerating research efforts to improve the lives of individuals affected by myotonic dystrophy and pave the way toward finding effective treatments and ultimately a cure for this challenging condition. There are multiple fantastic opportunities available - learn more below & get ready to apply!

March 29,2024

Meet the Early Career Grant Recipient: Dr. Johanna Hamel

Dr. Johanna Hamel, an Assistant Professor in Neurology, Pathology, and Laboratory Medicine, developed her interest in DM1 and DM2 during medical school in Germany. Her residency at the University of Rochester, chosen for its focus on neuromuscular medicine and DM, led to a fellowship under Dr. Charles Thornton, funded by the Clinical Research Training Fellowship Award in Muscular Dystrophy.

March 20,2024

Meet the DM Drug Developers

Leading up to and after the 2024 MDF Regional Conferences in the spring, our biotechnology and pharmaceutical partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions in real time.

February 14,2024

Research Fellow Feature: Cameron Niazi

After spending two years at Vertex Pharmaceuticals, Cameron Niazi pursued a Ph.D. at the University of Florida in Dr. Eric Wang's lab. Attracted by the lab's integration of basic science and translational research, joining the Wang Lab also provided a personal connection to DM1.

Questions?

Request Warmline Support! MDF offers a professionally staffed Warmline for people living with myotonic dystrophy, their caregivers, medical professionals and others to ask questions, find support and guidance and, when appropriate, be connected to additional resources and services. Click here to request Warmline Support.

Additional questions? Contact MDF by email at info@myotonic.org or give us a call at 415-800-7777.