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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

MDF is also 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.

Reduced MBNL1 Precedes Structural and Functional Changes in the DM1 Mouse Brain

A new DM1 mouse model, with postnatal expression of expanded CUG repeat RNA in the brain, implicates reduced MBNL1 and MBNL2 in the staging of pathological and functional changes.

Epigenetics Underlying the Parent of Origin Effect in CDM

Epigenetic modifications upstream of an expanded DMPK allele may underlie the maternal bias in the inheritance of CDM.

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