Myotonic Dystrophy Clinical Research Program

Our Mission

Our center has focused on Myotonic Dystrophy (DM) research and care for over 30 years. Our overarching goal is to find therapies for DM1 and DM2 that change people’s lives by treating the diverse symptoms of DM. We have several studies for children and adults, including some that you can do in your home without traveling. 

Why UR – What Sets Us Apart

At UR, we work as a team of scientists, clinicians, and physical therapists, covering the spectrum of research from "the bench to the bedside". Areas of expertise include outcome measures, molecular biomarkers, patient-reported outcomes, remote assessments, patient registries, and clinical trials. 

Our Clinical Research Program currently entails the following active projects:

• The REAL Lab: Remote Research Laboratory
• Women’s health studies
• Studies to develop biomarkers
• Exercise interventions
• The National Registry: to connect patients with researchers, and learn how symptoms change over time
• Clinical Trials (ongoing trials include Harbor and Harbor OLE and Freedom DM1)