Research Fellow Profile: Dr. Ginny Morriss

Ginny Morriss, Ph.D., is exploring whether reducing levels of the CELF1 protein, which are abnormally high in DM1-affected skeletal muscles, has a positive effect on these muscles. She’ll be studying mice in which disease-causing repeat expansions can be induced at any age.

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.

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.

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). 

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

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