Common Antibiotic Might Help Treat Myotonic Dystrophy Type 1 – Promising Therapy

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In a study published in December 2015 in a peer review journal researchers from Japan and Poland found that a commonly used antibiotic might assist in the treatment of Myotonic Dystrophy. This is a sort of stunning discovery as there is no treatment identified to treat the disease. Treatment now consists of reducing symptoms.

The researchers first began by screening antibiotics. In a screen of 20 antibiotics 2-3 were found to have some potential with the disease.When screening the drugs they first used mice cells and lab equipment to find the most promising compounds (drugs). . Erythromyicin was found to have the highest attraction to the RNA CUG expansion (The opposite of CTG repeats in the DNA) Erythromycin was the drug that the researchers chose to study. Click here for the screening graph Muscleblind and Various antibiotics and compounds

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Florida Funding attract Key Myotonic Dystrophy Researchers to University of Florida

Building a Preeminent Research Program: The Center for NeuroGenetics

As an introduction to this newsletter, please check out a video created by Eric Wang, Ph.D., who is joining UF as an assistant professor of molecular genetics and microbiology in the Center for NeuroGenetics. This edition of On The Same Page is the story of how new and existing faculty can come together to create preeminent research programs.

In the UF community, the concept of “preeminence” has been used quite a bit of late. Florida’s “Preeminence Bill,” signed into law during the 2013 legislative session, designated the University of Florida as a preeminent university in the state. To help UF attain its goal of becoming a top 10 public university, a section of this bill provides funds to UF for the purpose of attracting outstanding faculty to advance our research mission.

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Cold Weather and Myotonic Dystrophy

COLD WEATHER and MYOTONIC DYSTROPHY

There is no definitive information on cold weather and it’s effects on patients with myotonic dystrophy vary from person to person. Some individuals have reported problems, and here is some information collected from various resources. Its clear that for some individuals there is a mild moderate effect, for others its more severe. If possible plan long term to move to a more temperate climate without harsh winters.

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Myotonia Drugs to Help prevent Muscle Contractures and Seizures

Here is some information about a drug that may help with muscle contractures and seizures. My son recently had several episodes where he would lose balance and sink to the ground, not really tripping but losing strength and we suspected that he may be having myotonia contractures. What this is simply is that muscles in his legs are seizing up and not moving so that the body can not remain upright. We are looking at some of these drugs to see if they may be helpful. The one that has been mentioned most is mexiletine. There are a couple others but Dr. Moxley at the University of Rochester has studies this drug more than most. His experience with Myotonic Dystrophy is extensive.

The study did not measure outcomes. That is they studies it and saw it reduced ontraction time in patients muscles. They did not look to see if it made a difference in their everyday lives such as walking or opening cans. So no real evidence it helps outcomes, but it seems to work from the researchers standpoint.

The study here was presented at one of the IDMC meetings. Will be giving this drug a try if the doctors agree it may help and not affect any of his cardiac issues, like level 1 heart block.

 

Mexiletine is an effective antimyotonia treatment in myotonic dystrophy type 1(LOE Classification)

This article has been cited by other articles in PMC.

Abstract

Objective:

To determine if mexiletine is safe and effective in reducing myotonia in myotonic dystrophy type 1 (DM1).

Background:

Myotonia is an early, prominent symptom in DM1 and contributes to decreased dexterity, gait instability, difficulty with speech/swallowing, and muscle pain. A few preliminary trials have suggested that the antiarrhythmic drug mexiletine is useful, symptomatic treatment for nondystrophic myotonic disorders and DM1.

Methods:

We performed 2 randomized, double-blind, placebo-controlled crossover trials, each involving 20 ambulatory DM1 participants with grip or percussion myotonia on examination. The initial trial compared 150 mg of mexiletine 3 times daily to placebo, and the second trial compared 200 mg of mexiletine 3 times daily to placebo. Treatment periods were 7 weeks in duration separated by a 4- to 8-week washout period. The primary measure of myotonia was time for isometric grip force to relax from 90% to 5% of peak force after a 3-second maximum grip contraction. EKG measurements and adverse events were monitored in both trials.

Results:

There was a significant reduction in grip relaxation time with both 150 and 200 mg dosages of mexiletine. Treatment with mexiletine at either dosage was not associated with any serious adverse events, or with prolongation of the PR or QTc intervals or of QRS duration. Mild adverse events were observed with both placebo and mexiletine treatment.

Conclusions:

Mexiletine at dosages of 150 and 200 mg 3 times daily is effective, safe, and well-tolerated over 7 weeks as an antimyotonia treatment in DM1.

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Staying upright with a Neuromuscular disease like Myotonic Dystrophy

Staying Upright with a Neuromuscular Disease

From: Multiple Sclerosis Society Quest newsletter, written by Christan Medvescek

Some people have nightmares about falling off cliffs. Brad Williams has nightmares about falling — period.

“Whenever I’m walking, falling down is always the major thought on my mind,” says Williams, 39, of Alexandria, Va. “It has to be on my mind a lot for me to be dreaming about it.”

Williams has Miyoshi distal myopathy, a slowly progressive form of muscular dystrophy that primarily affects the extremities. He hosts an MDA Internet chat under the nickname “dysf,” and notes that other chat participants also have reported falling nightmares. “It’s like a flying-falling dream, except it’s just about falling down.”

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