the 2017 Muscle Study Group Annual Meeting
was held September 23-25, 2017, at the Snowbird Ski and Summer Resort in Snowbird, Utah.
Link to the abstracts below:
https://onlinelibrary.wiley.com/doi/10.1002/mus.25768
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
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
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
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.
The purpose of this study is to evaluate the efficacy and tolerability of prednisone in patients diagnosed with ocular myasthenia. This trial is currently recruiting participants.
Sponsor: Michael Benatar
The purpose of this study is to determine whether Hizentra is a safe and effective treatment for people with myasthenia gravis (MG).
Sponsor: Mazen Dimachkie, MD
Year MSG Membership Ends: 2022
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
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
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:
The purpose of this trial is to determine if thymectomy combined with prednisone therapy is more beneficial in treating non-thymomatous myasthenia gravis than prednisone therapy alone.