$700,000 New Grant Award

MDF, in partnership with MDF UK and other donors, has announced $700,000 in funding to support a new natural history and genetic modifier study at sites in the Myotonic Dystrophy Clinical Research Network (DMCRN), and to expand the number of sites included in the Network. The DMCRN is a network focused on conduct of clinical research studies. The six initial DMCRN sites have completed a 100-patient natural history study that has helped identify potential clinical trial and study biomarkers and has informed the design of trials of candidate therapeutics for DM.

DMCRN Expands to 9 Sites

The DMCRN has expanded to 9 sites through new funding from MDF and MDF UK, to include the University of Utah, Houston Methodist Neurological Institute, and the University of Iowa. The DMCRN will soon launch a 500-patient natural history study with unrestrictive entry criteria, ensuring that virtually any person with DM1 is included. This new study will examine a larger number of patients (500) over a longer time period (2 years) that will allow identification of clinical trial outcome measures best suited to testing new candidate drug and biological therapies. The success of clinical trials hinges on identifying outcome measures that are sensitive, show changes during the typical 6 to 12 month duration of a trial, and reflect activities that are meaningful to the patient. Natural history studies are essential in identifying such outcome measures.

The new DMCRN natural history study, in recruiting 500 subjects, is ambitious. DMCRN leadership has been intent on expanding the network to improve patient access to participating sites and to help meet this ambitious goal. Study leadership’s focus has always been on addition of only well-qualified sites.

MDF is pleased to welcome the Universities of Iowa and Utah, and the Houston Methodist Neurological Institute to the DMCRN. Drs. Tetsuo Ashizawa, Laurie Gutmann, Nick Johnson and Peg Noupoulos have significant experience in DM research and clinical care programs. Their expertise and additional DM patient populations will help the DMCRN meet its recruitment goals and thereby improve clinical trial readiness for DM.