Using PROMS to Evaluate Potential Therapies

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.

The Curious Dilemma of Classifying DM1

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.

Internuclear Transfer of Toxic RNA in DM1?

An improved understanding of the mobility of toxic RNA in DM1 has come from muscle precursor transplantation studies.

Small Molecule Candidates Targeting Primary Disease Mechanisms in DM

A new review article looks at efforts to target DM disease mechanisms with small molecule compounds.

Toward Functional Outcome Measures for Clinical Trials in DM1

Steps are taken to help resolve the adequacy of functional outcome measures for interventional clinical trials in DM1.

Dmpk Silencing is Unlikely to be a Confounding Factor for ASO Treatment of DM1

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

Gender-Related Cancer Risk in DM1

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). 

New Grants Awarded

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.

New Study on DM1 Brain Changes

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.

New Grants Drive DM Research

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.

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