DM1

Molecular Events Underlying Congenital DM

A new study points to the timing of MBNL-dependent RNA processing defects as a major factor in the pathogenesis of CDM.

Understanding and Measuring Fatigue in DM1

Although fatigue represents a substantial burden in DM1, tools must be validated to assess its diverse contributing factors in order to develop clinical trial endpoints and effective therapies.

Preclinical Data Behind the Ionis Trial Published

Preclinical animal efficacy data used to support the scientific rational for Ionis’ phase 1/2 clinical trial in DM1 was just published.

New Drosophila Models for DM1 and DM2

New fly models show a DM2 phenotype at least as severe as DM1 and may provide a platform for studies of genetic modifiers and candidate therapy screening in DM.

DM1 Genotype and Cardiac Phenotype

A large retrospective study helps clarify genotype-cardiac phenotype relationships in adult DM1.

Do We Treat DM as a Brain Disease?

A recent review article makes the case that DM is a brain disease and that better understanding of and treatment strategies for the neurological consequences of DM are essential.

DM Highlights at American Academy of Neurology Annual Meeting

MDF staff recently attended the 2017 annual meeting of the American Academy of Neurology, in Boston, MA. Here are highlights from that meeting.

Inheritance of CDM

There have been new discoveries in the way that congenital myotonic dystrophy (CDM) is inherited.

Telling the Quacks from the Cures

Myotonic is happy to help you understand whether something is in a legitimate clinical trial, an approved therapy…or not.

Patient-Reported Data to Guide Care and a Cure for DM

The UK DM Patient Registry reports on disease burden for 556 patients with a confirmed diagnosis of DM1.

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