MSG Annual Meeting Archives
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:
NuFactor, Inc.
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 GuptillA 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
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
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
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 MG –Michael 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 CMT1A –Michael 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.
