2016 MSG Annual Meeting

The 2016 Muscle Study Group annual Meeting was held September 24-26, 2016, at the Snowbird Ski & Summer Resort in Snowbird, UT. Please click on the following links to be directed to the presentation slides, Poster Abstracts, and agenda for the meeting.

Final Agenda

Abstracts: Link to Abstracts

Presentations: Link to Presentations

Sponsors

MSG 2016 Sponsors

Natural History Study of Spinal Muscular Atrophy (SMA)

Spinal Muscular Atrophy (SMA) is the leading genetic cause of death in infancy. It is a devastating disease that leads to progressive loss of those nerve cells that control our muscle bulk and movement. Patients develop increasing weakness in all muscles, eventually including those needed for breathing. In more than half of patients, SMA starts in infancy and typically leads to death within the first 2 years of life. In others, the disease begins in childhood and leads to significant disability.

SMA is caused by a defect in the “Survival of Motor Neurons” (SMN1) gene. Researchers are hopeful to find a cure, because nature has provided humans with a second gene, almost an identical copy of the SMN1 gene. Normally, the second gene does not contribute much, but researchers think that its function can be increased by medications.

To find out whether these medications help patients with SMA, we have to conduct clinical trials. Here, we propose to prepare for clinical trials. We will invite SMApatients to join our research effort. We will examine them regularly to better understand their disease. The visits will include questions, physical exam, blood drawing, and sometimes X-rays and a skin biopsy. We will use modern computer methods to process the information. While we are doing this, we will plan a clinical trial. Once the clinical trial begins, we will offer SMA patients participation if they meet the criteria for that trial.

We will make sure that the participants’ privacy is maintained and that the study risks are as low as possible.

Identifying an effective SMA treatment is very important because there is currently none. Clinical trials are the only way to decide whether a new treatment works in SMA patients or not.

Sponsor: Columbia University

Ascorbic Acid Treatment of Charco-Marie Tooth Type 1A (CMT)

This study will look at the impact of ascorbic acid (Vitamin C) on the progression of disease in people with CMT1A as compared to volunteers receiving a placebo. This study will assess whether is it futile to proceed with a larger, longer-term, placebo-controlled study.

Sponsor: Wayne State University

Dichlorphenamide vs. Placebo for the Treatment of Patients with Hyperkalemic or Hypokalemic Periodic Paralysis (HYP-HOP)

The purpose of this study is to compare Dichlorphenamide with placebo (an inactive substance) for prevention of episodes and for improvement of strength in hyperkalemic (HYP) and hypokalemic (HOP) periodic paralysis. This study will also look at the long-term effects of Dichlorphenamide in periodic paralysis.

Sponsor: University of Rochester
This trial no longer recruiting.

Methotrexate in Myasthenia Gravis (Methotrexate)

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

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)

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