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Inheritance of CDM

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

Telling the Quacks from the Cures

Many of you have seen posts on social media about treatments they have received in other countries or heard about through friends, that include everything from dietary aids to gene therapy, and want to how you can assess the possible benefits and risks of these 'treatments.' In this complicated therapy environment, how can patients make decisions about whether an available treatment or therapy is safe and effective? How can you tell the quacks from the cures?

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.

A Biomarker for Cardiac Dysfunction in DM1?

A multi-center study suggests serum cardiac troponin-1 levels predict risks of left ventricular dysfunction in DM1 patients.

Using Gene Editing to Correct DM

A potentially revolutionary technology may allow development of a drug for DM that can correct a patient’s DNA by selectively removing the expanded CTG and CCTG repeats in DM1 and DM2, respectively.

Gene Editing for DM

Gene editing is a potential avenue for therapy development in DM. With the safety, efficacy and delivery challenges, how do we get there?

Improving Clinical Trials in Myotonic Dystrophy: Thurman Wheeler, M.D.

When Dr. Thurman Wheeler was a resident in neurology, he remembers a senior physician telling him that myotonic dystrophy would probably be one of the most difficult diseases to treat because it involves so many body systems.

Treating Sleep Disorders in DM1

Sleep studies are indicated for DM1 patients with disrupted sleep or daytime sleepiness in order to direct selection of the optimal treatment regimen.

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