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.


The Muscle Study Group received permission from the authors to post the following presentations online.

Supporting the Discoveries of TomorrowSanjay Bidichandani

Development and Validation of the MG Composite and MG-QOL15Ted Burns

Adaptive Clinical Trial DesignsScott Evans

Recent Developments in SMAJohn Kissel

Functional MeasuresJackie Montes

Rituxan in Myositis Study – the Tale of a Novel Trial DesignChester Oddis

Outcome Measurements as Clinical BiomarkersSindhu Ramchandren

Electrical Impedance MyographySeward Rutkove


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 KingdomLiz 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 RegistryTodd Levine

Johns Hopkins Myositis Center Experience with Inclusion Body MyositisThomas 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 StudyPedro Machado

Antibody Levels Correlate with Creatine Kinase Levels and Strength in Anti-HMG-CoA Reductase-Associated Autoimmune MyopathyAndrew Mammen

CLCN1 Mutation is Acting as a Modifier Gene in an Italian DM2 Family with Juvenile OnsetGiovanni Meola

MRI Quantification of Abnormal Muscle Water Distribution in Chronic Neuromuscular Diseases: A Sensitive BiomarkerJasper Morrow

Efficacy and Safety of Privigen in Patients with Chronic Inflammatory Demyelinating Polyneuropathy: The PRIMA TrialPatty Riley

Exploring Activity Levels in People with Charcot-Marie-Tooth Disease or Inclusion Body Myositis in Comparison to Healthy ControlsAmanda Wallace

Hereditary Spastic Paraplegia Genetics in the Exome Era: Data on 123 HSP ExomesStephan Zuchner


The Muscle Study Group would like to thank the following sponsors for the 2012 MSG Annual Meeting:


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