Methotrexate in Myasthenia Gravis (Methotrexate)

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Myasthenia gravis is a rare neuromuscular disorder characterized by weakness and fatigability of ocular, bulbar, and extremity musculature. The specific aim of this study is to determine if oral methotrexate is an effective therapy for myasthenia gravis (MG) patients who are prednisone dependent. Patients will be randomized to receive either methotrexate or placebo and those who are entered onto this trial will have symptoms and signs of the disease while on prednisone therapy. The hypothesis is that adding methotrexate therapy in these patients will improve the MG manifestations so that the prednisone dose can be reduced and clinical measures of MG severity will improve.

Sponsor: University of Kansas Medical Center

2015 MSG Annual Meeting

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MSG Attendee Picture

 

The 2015 the Muscle Study Group Annual Meeting took place at its new home in Snowbird, UT at the Snowbird Ski & Summer Resort.

This year’s meeting, titled “Experimental Therapeutics Across the Spectrum of Neuromuscular Disease” was a huge success and brought together members from all across the globe, including pharmaceutical industry, NIH and academic researchers.

Final Agenda

2015 MSG Meeting Final Agenda

Abstracts

Meeting abstracts were published in Muscle & Nerve 2015; 52:S1 (September 2015)

Presentations

The Muscle Study Group has received approval from the authors to publish the following presentations on our website:

An Ounce of Prevention is Worth a Pound of Cure; Michael P. McDermott, PhD

NINDS Funding Opportunities; Glen Nuckolls, PhD

Placebo, History and Impact on Clinical Trials Outcome; Aziz Shaibani, M.D.

Best Practices for Preclinical Research in Neuroscience; Oswald Steward, PhD

Cost of Drugs for Neuromuscular Disorders; Donald B. Sanders, M.D.

Clinical Trials Programs at NIAMS; Tom Cheever, PhD

Sponsors 

The Muscle Study Group wishes to thank the sponsors of the 2015 annual meeting:

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Mexiletine and Non Dystrophic Myotonias (MYOMEX)

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Treatment strategies in non-dystrophic myotonias are based on selective case reports, clinical experience and theoretical benefit. Presently, the most promising antimyotonic medication is mexiletine (MEX) but its manufacturing was stopped. The proposed randomized, double-blind, placebo-controlled, crossover trial is designed to:

  1. study the safety and efficacy of mexiletine for the treatment of non-dystrophic myotonias
  2. validate electromyographic tests as a standardized outcome measure of myotonia
  3. assess the reliability and validity of a new clinical rating scale for myotonia

Characteristics of Episodic Ataxia Syndrome

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Episodic ataxia (EA) is a rare genetic disease characterized by episodes of imbalance, incoordination, and slurring of speech. The underlying cause of EA is only partly understood, and currently there are no established treatments. There is also little information about the link between EA’s clinical features and its genetic basis. The purpose of this study is to better characterize EA and disease progression. In turn, this may direct the development of future treatments.

 

Effect of Potassium and Acetazolamide on People With Andersen-Tawil Syndrome

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Andersen-Tawil Syndrome (ATS) is a rare genetic disorder that causes episodes of muscle weakness, potentially life-threatening changes in heart rhythm, and skeletal developmental abnormalities. The cause of some ATS cases remains unknown, and no specific treatments have been established. The purpose of this study is to determine whether potassium supplements and/or the medication acetazolamide affect the duration of muscle weakness and heart rhythm abnormalities in people with ATS.

 

Characteristics of Andersen-Tawil Syndrome

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Andersen-Tawil Syndrome (ATS) is a rare, genetic disorder that causes episodes of muscle weakness, potentially life-threatening changes in heart rhythm, and developmental abnormalities. Disease symptoms can vary, the cause of some ATS cases remains unknown, and no specific treatment has been identified. The purpose of this multi-site study is to better characterize ATS, establish whether symptoms change over time, and determine if symptoms are related to a mutation in the KCNJ2 gene.

 

Consortium for Clinical Investigation of Neurologic Channelopathies (CINCH)

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Welcome to the Consortium for Clinical Investigations of Neurological Channelopathies

Why am I here?
This consortium exists to bring together physicians and patients for the sake of Neurological Channelopathies research.

What can I do?
Take Action. Learn more about joining our registry. Research offers no guarantees, but one thing is for certain, research cannot move forward with out your help. Every active role a patient takes may possibly play a part in discovering new groundbreaking research and finding new treatments.