The MSG held its annual meeting on September 27-29, 2012, at Beaver Hollow Conference Center, Java Center, NY.
The meeting, entitled “Clinical Trial Design and Biomarkers in Neuromuscular Disease,” brought together members of the pharmaceutical industry, NIH, FDA and academic researchers.
The agenda included five major sessions: Clinical Trial Design: Novel Approaches, NIH and Foundation Perspectives, Overview of Biomarkers as Indicators of Therapeutic Efficacy and the FDA’s Perspective, Pathogenesis and Treatment: Recent Discoveries, and Update of Specific Biomarkers as Indicators of Therapeutic Efficacy in Neuromuscular Disease.
Presentations
The Muscle Study Group received permission from the authors to post the following presentations online.
Supporting the Discoveries of Tomorrow – Sanjay Bidichandani
Development and Validation of the MG Composite and MG-QOL15 – Ted Burns
Adaptive Clinical Trial Designs – Scott Evans
Recent Developments in SMA – John Kissel
Functional Measures – Jackie Montes
Rituxan in Myositis Study – the Tale of a Novel Trial Design – Chester Oddis
Outcome Measurements as Clinical Biomarkers – Sindhu Ramchandren
Electrical Impedance Myography – Seward Rutkove
Abstracts
Abstracts for which approval was received to post online are listed below.
Strength and Functional Outcomes in LGMD2a – Lindsay Alfano
Frequency and Circumstances of Falls in People with Inclusion Body Myositis in the United Kingdom – Liz Dewar
Are Sensory Electrodiagnostic Parameters Useful in Differentiating Axonal and Demyelinating Neuropathies? – Raghav Govindarajan
Rates of Adverse Events After High Dose IVIG Infusions Do Not Differ Among Patients Receiving 2 Day versus 5 Day Infusions – Shafeeq Ladha
ADAPT: Acthar in Dermatomyositis and Polymyositis Treatment Registry – Todd Levine
Johns Hopkins Myositis Center Experience with Inclusion Body Myositis – Thomas Lloyd
Development of a Virtual Upper Extremity Assessment Tool for Individuals with DMD Across the Lifespan – Linda Lowes
Natural History of Sporadic Inclusion Body Myositis — An Observational Longitudinal Study – Pedro Machado
Antibody Levels Correlate with Creatine Kinase Levels and Strength in Anti-HMG-CoA Reductase-Associated Autoimmune Myopathy – Andrew Mammen
CLCN1 Mutation is Acting as a Modifier Gene in an Italian DM2 Family with Juvenile Onset – Giovanni Meola
MRI Quantification of Abnormal Muscle Water Distribution in Chronic Neuromuscular Diseases: A Sensitive Biomarker – Jasper Morrow
Efficacy and Safety of Privigen in Patients with Chronic Inflammatory Demyelinating Polyneuropathy: The PRIMA Trial – Patty Riley
Exploring Activity Levels in People with Charcot-Marie-Tooth Disease or Inclusion Body Myositis in Comparison to Healthy Controls – Amanda Wallace
Hereditary Spastic Paraplegia Genetics in the Exome Era: Data on 123 HSP Exomes – Stephan Zuchner
Sponsors
The Muscle Study Group would like to thank the following sponsors for the 2012 MSG Annual Meeting:
