MSG Annual Meeting Archives

2013 MSG Annual Meeting

The 2013 Muscle Study Group Meeting was held at Lady Margaret Hall, Norham Gardens, Oxford, UK - September 16, 17 and 18.

This year's meeting was entitled Novel Molecular Mechanisms of Neuromuscular Disease: Implications for Therapy brought together members of the pharmaceutical industry, NIH, FDA and academic researchers.

Final Agenda

Please see: Final Agenda

Group Photo

2013 MSG Meeting Group Photo

Abstracts

Meeting abstracts were published in Muscle and Nerve 2013;48:S1 (September 2013).

Sponsors

We wish to thank the following for their support of this year's meeting:

CSL Behring Biotherapies for Life-HomeNuFactorGenzyme Logo

MDA Logo NIH Logo

cid:image001.jpg@01CD602A.A56A4330 Valerion LogoWalgreens



2012 MSG Annual Meeting

The MSG held its annual meeting on September 27-29, 2012 at Beaver Hollow Conference Center, Java Center, NY.
This year's 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: (1) Clinical Trial Design: Novel Approaches, (2)  NIH and Foundation Perspectives , (3) Overview of Biomarkers as Indicators of Therapeutic Efficacy and the FDA's Perspective, (4)Pathogenesis and Treatment: Recent Discoveries and (5) Update of Specific Biomarkers as Indicators of Therapeutic Efficacy in Neuromuscular Disease.

 

This meeting was sponsored by:

Athena LogoATG Rehab LogoCSL Behring Biotherapies for Life-HomeMDA LogoWalgreensQuestcor Pharmaceuticals cid:image001.jpg@01CD602A.A56A4330

NuFactor

 

MSG 2012 Group Photo

 

Presentations for which approval was received to post on the internet are below:

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 for which approval was received to post on the internet are 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

 

 

2011 MSG Annual Meeting

The MSG held its annual meeting on September 19-21, 2011 at Beaver Hollow Conference Center, Java Center, NY.
This year's meeting entitled “Translational Research in Neuromuscular Diseases: Diverse Diseases, Convergent Themes ” brought together members of the pharmaceutical industry and academic researchers.

The agenda included five major sessions: (1) RNA Metabolism as Therapeutic Target in Muscle Disease, (2)  RNA Metabolism as Therapeutic Target in Motor Neuron Disease , (3) Government/Foundation Perspectives on NM Themes, (4) Animal Models and Translational Researchand (5) Exercise as a therapy for NM Disease.

 

This meeting was sponsored by:

  • National Institutes of Health (NIH) Grant #1R13NS076196-01 
  • Muscular Dystrophy Association
  • ISIS Pharmaceuticals
  • PTC Therapeutics
  • Crescent Healthcare, Inc.
  • Genzyme Corporation

MSG 2010 Group Photo
Presentations for which approval was received to post on the internet are below:

Abstracts for which approval was received to post on the internet are below:

Fat Metabolism Syndrome in Patients with DMD Following Low Energy Femur Fracture - Emma Ciafaloni

Plasma Exchange Complications are Related to the Venous Access Route - Jeffrey Guptill

A Prospective, Randomized Controlled Trial of valproic Acid in Ambulant Adults with SMA: The VALIANT Trial - John Kissel

Amyloidosis and Exercise Tolerance in AN05 Muscular Dystrophy - Margherita Milone

MRI Quantification of Lower Limb Muscle Fatty Atrophy: A Potential Outcome Measure in Chronic NM Diseases - Jasper Morrow
Identifying the Cause of Phenotypic Variability in a Family with NDM- Dips Raja Rayan

2010 MSG Annual Meeting

The MSG held its annual meeting on September 20-22, 2010 at Beaver Hollow Conference Center, Java Center, NY.

The agenda included five major sessions: (1) The Challenges of Patient Recruitment, (2)  Recruiting the Next Generation of NMD Investigators, (3) Inflammatory Myopathies: Pathogenesis, Treatment and Clinical Trials, (4) Inflammatory Myopathies: Pathogenesis, Treatment and Clinical Trials (Continued) and (5) Past, Current and Future Protocols: Lessions Learned.

 

This meeting was sponsored by:

  • National Institutes of Health (NIH) Grant #1R13NS071695-01: 
    • National Institute of Neurological Disorders and Stroke (NINDS)
    • Office of the Director (OD)
    • National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
  • Muscular Dystrophy Association
  • ISIS Pharmaceuticals
  • PTC Therapeutics

MSG 2010 Group Photo

 

Presentations for which approval was received to post on the internet are below:

Myositis Patient Registry - Bob Goldberg

FOR-DMD Steroid Trial - Robert Griggs

European Perspective on Recruitment - Michael Hanna

Update from British Myology Society - Michael Hanna

Young Neurologists in the Basic Laboratory - Ahmet Hoke

NIH Report on Support for Research on Neuromuscular Diseases - Glen Nuckolls

IMACS History and Development - Chester Oddis

Abstracts for which approval was received to post on the internet are below:

Safety and Feasibility of Transvenous Limb Profusion with Saline in Human Muscular Dystrophy - James Howard

Mechanism of Axonal Degeneration in Drosophila Model of Distal SBMA - Thomas Lloyd

Reproducibility and Reliability of Quantitative MRI Measures for Neuromuscular Disease - Jasper Morrow

Clinical Improvement and Decreased Peripheral Autoreactivity in Refractory Myasthenia Gravis Treated with Rituximab - Richard Nowak

A Therapy for Duchenne Dystrophy Based Upon Inhibition of Mechanically Sensitive Ion Channels - Frederick Sachs

Inherited Neuropathies Consortium - Carly Siskind

 

2009 MSG Annual Meeting

The MSG held its annual meeting on September 21-23, 2009 at Beaver Hollow, Java Center, NY.
This years meeting entitled “Fostering Pharma-Academic Partnerships in Neuromuscular Experimental Therapeutics” brought together members of the pharmaceutical industry and academic researchers.

The agenda included two major sessions: (1) Novel designs and outcome measures in translational neuromuscular research and (2)  scientific updates in basic science, methods and treatment strategies.

This meeting was sponsored by:

  • National Institutes of Health (NIH): 
    • National Institute of Neurological Disorders and Stroke (NINDS)
    • Office of the Director (OD)
    • National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
  • Muscular Dystrophy Association
  • Genzyme Corporation
  • ISIS Pharmaceuticals
  • PTC Therapeutics
  • Santhera Pharmaceuticals

Presentations for which approval was received to post on the internet are below:

The Peripheral Nerve Study Group - Its Rise and Demise -Arthur Asbury

Treatment of Neuromuscular Diseases With Antisense Oligonucleotides - Frank Bennett

FOR - DMD - Kate Bushby

MSG - Past, Present, Future - Robert Griggs

HYP-HOP - Robert Griggs

The SMA Project - Jill Heemskerk

High Dose Vitamin C Treatment of CMT CMT-1A - Richard Lewis

Dermal Nerve Assessment: A Window to the Mechanism of Neuropathies in Outcome Measures - Jun Li

The Challenges of Conducting Clinical Development in Rare / Orphan Diseases: The Industry Perspective - Thomas Meier

Attaining Symbiosis in Therapy Development Efforts in NMDs - John Porter

Trial Design and Outcomes for Inherited Neuropathy Studies - Davide Pareyson

Abstracts for which approval was received to post on the internet are below:

TREAT-NMD and the ICC: A Fruitful Collaboration to Foster Clinical Trials and Research in SMA - Anna Ambrosini

Using Video Gait Analysis to Assess Gait Dysfunction in Duchenne Muscular Dystrophy: A Pilot Study - Yaacov Anziska

Use of the 6-Minute Walk Test to Assess Potential Drug Effects of Ataluren (PTC124™) in Duchenne/Becker Muscular Dystrophy - Leone Atkinson

Mechanostat theory used to understand bone complications in children with Duchenne muscular dystrophy - Craig Campbell

Sparing of Myofiber Membranes in Infantile Pompe’s Disease - Ludwig Gutman

Clinical development of idebenone (CATENA®) for the treatment of Duchenne Muscular Dystrophy – A summary of Phase II study results and design of a Phase III clinical study - Thomas Meier

The challenges of conducting clinical development in rare / orphan diseases – The industry perspective - Thomas Meier

Diagnosis of Pompe Disease: Timing and Methods Used as Reported to the Pompe Registry - Tim Miller

Fatty Acid Oxidation Disorders in Adults: A potentially treatable cause of muscle disease - Robert Pitceathly

Presence and Nature of “Tau” Immunoreactivity in Normal Myonuclei and Inclusion Body Myositis - Mohammad Salajegheh

Initial Results from Single Subcutaneous Administration of ACE-031, a Form of the Soluble Activin Type IIB Receptor, in Healthy Postmenopausal Volunteers - Jasbir Seehra

 

2008 MSG Annual Meeting

The MSG held a meeting of clinical scientists on September 15-17, 2008 at Beaver Hollow, Java Center, NY entitled “Experimental Therapeutics of Neuromuscular Disease”. The conference brought together translational and clinical scientists in a forum for discussion of current and novel therapies and drew speakers and participation from international experts with an interest in developing international collaborations between trainees and investigators focused on clinical trials. Specific to international collaboration many members of the TREAT-NMD consortium based in Europe attended the meeting thereby fostering connections necessary to facilitate clinical trials in the rare diseases.

The agenda included two major sessions: (1) Novel designs and outcome measures in translational neuromuscular research and (2)  scientific updates in basic science, methods and treatment strategies.

This meeting was sponsored by:

  • National Institutes of Health (NIH): 
    • National Institute of Neurological Disorders and Stroke (NINDS)
    • Office of the Director (OD)
    • National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
  • Muscular Dystrophy Association
  • Acceleron Pharma

Presentations for which approval was received to post on the internet are below:

Ocular MGMichael Benatar 

Sleuthing Targets for Disease in Muscle - Andrew Engel

Clinical Investigation of Neurological Channelopathies
(CINCH) Landmarks
- Robert C. Griggs

HYP HOP - Robert C. Griggs

Clinical Trials: From Inception to Implementation - Laura Herbelin

Trial Implementation Strategies - Cornelia Kamp

Clinical Materials Services Unit (CMSU) - Cornelia Kamp

Iplex Treatment of Myotonic Dystrophy - Richard T. Moxley, III

High Dose Ascorbic Acid to Treat CMT1AMichael Shy

 

MSG 2007Annual Meeting

Through the funding of a NIH R13 award the MSG held a meeting of clinical scientists on September 17-19, 2007 at Beaver Hollow, Java Center, NY. The program emphasized translational approaches to bringing molecular discoveries into well-designed clinical studies.

The agenda included two major sessions: (1) Novel designs and outcome measures in translational neuromuscular research and (2)  scientific updates in basic science, methods and treatment strategies.

 

MSG 2006 Annual Meeting

Through the funding of a NIH R13 award MSG held a meeting of clinical scientists on May 31 - June 2, 2006 at Beaver Hollow, Java Center, NY. The program emphasized translational and experimental therapeutics research.

The agenda and included two major sessions: (1) Translational bench-to-bedside presentations on topics including muscular dystrophy, neuromuscular junction disorders and motor neuron disorders and (2) Clinical trial methodology session including industry, FDA and academic perspectives.