DM1

Scientists from a consulting firm with considerable experience in evaluation of patient-reported outcome measures evaluated available PROMs for their potential in DM1 clinical trials.

Analysis of data from the French DM-Scope registry lend support to a five-grade model of DM1 that may help guide patient management, biomarker and modifier gene discovery, and clinical trials.

New studies in mouse models show that constitutive or acquired loss of Dmpk has no effects on skeletal or cardiac muscle function.

A recent study corroborated increased susceptibility to cancer in DM1, for women in particular, and linked the elevated risk to depressed levels of a tumor suppressor microRNA (miRNA). 

MDF has awarded new grants to develop phenotypic mouse models of DM as well as new DM1 and DM2 cell lines. Read more about these new partnerships.

A new study from Dr. Dimitri Renard and his team applies metabolic imaging to identify brain regions affected in DM1. Read about their findings here.
 

MDF and the MDF UK jointly announce the funding of two new research projects. The projects address critical gaps in research infrastructure and clinical trial readiness and will increase understanding of the progression of DM, and provide measures to evaluate disease progression and the efficacy of candidate therapeutics.