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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.

Qualifying a Biomarker for DM

A recent publication suggests that the DM field may be on the cusp of having a critically important molecular biomarker to facilitate decision-making in early-stage clinical trials.

Professionals Track at MDF Annual Conference a Success

For the first time, the 2016 MDF Annual Conference included a closed, professionals-only track focused on providing updates on basic scientific discovery and efforts focused on therapy development. 

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.

Kidney Dysfunction is a Risk in DM1: How to Best Assess It

Since DM1-related loss of muscle mass confounds a common biomarker of kidney dysfunction, creatinine levels, serum cystatin C provides a superior measure of renal dysfunction for both patient management and clinical trials.

 

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). 

Congenital and Juvenile-Onset Clinical Trial Launches

AMO Pharma Limited (AMO Pharma) today announced initiation of a phase 2 clinical study in the U.K. for AMO-02 (tideglusib).

Research Fellow Profile: Dr. Benjamin Gallais

Dr. Gallais and his team are conducting a longitudinal study of cognition and central nervous system involvement in DM1. Read an interview with Dr. Gallais on this work here.Highlights from IDMC-10

New Grants Awarded

MDF and the Wyck Foundation have 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.
 

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