2016 MSG Annual Meeting

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

MSG 2016 Sponsors

2015 MSG Annual Meeting

MSG Attendee Picture

 

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

2015 MSG Meeting 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:

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2014 MSG Annual Meeting

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The 2014 Muscle Study Group Meeting took place at the Beaver Hollow Conference Center (Java Center, NY) – September

The meeting, titled “Economic Considerations in the Development of New Treatments for Neuromuscular Diseases” brought together members of the pharmaceutical industry, NIH and academic researchers.

Final Agenda

2014 MSG Meeting Final Agenda

Abstracts

Meeting abstracts were published in Muscle and Nerve 2014;50:S1 (September 2014)

Presentations

The Muscle Study Group has received approval from the authors to publish the following presentations on our website.

Transcriptome Profiling Identifies Key Differences Between Patients with Recessive or Dominant Negative Mutations in Collagen VI Related MyopathiesRussell Butterfield

Complications of Pregnancy in Myotonic DystrophyNicholas Johnson

Funding New Investigators: The Role of the CTSAs Karl Kieburtz

Targeting Mitochondrial Free Radical Production in Aging Skeletal MuscleDavid Marcinek

Outcomes: MDA-NINDS WorkshopJohn Porter

Cataloging FDA’s Flexibility in Subpart H and Orphan Drug ApprovalsFrank Sasinowski

Patient-Reported Outcomes (PROs) in Randomized Clinical Trials (RCTs)Ira Shoulson

MRI in Experimental Muscle TherapeuticsTracey Willis

Sponsors

The Muscle Study Group wishes to thank the sponsors of the 2014 annual meeting:

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2013 MSG Annual Meeting

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

The meeting was entitled “Novel Molecular Mechanisms of Neuromuscular Disease: Implications for Therapy” and brought together members of the pharmaceutical industry, NIH, FDA and academic researchers.

Final Agenda

2013 MSG Annual Meeting Final Agenda

Abstracts

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

Sponsors

The Muscle Study Group wishes to thank the following for their support of the 2013 meeting:

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2012 MSG Annual Meeting

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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 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

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

Sponsors

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

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2011 MSG Annual Meeting

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

The agenda included five major sessions: RNA Metabolism as Therapeutic Target in Muscle Disease, RNA Metabolism as Therapeutic Target in Motor Neuron Disease, Government/Foundation Perspectives on NM Themes, Animal Models and Translational Research, and Exercise as a Therapy for NM Disease.

Abstracts

The Muscle Study Group received permission to post the following abstracts online.

Fat Metabolism Syndrome in Patients with DMD Following Low Energy Femur FractureEmma Ciafaloni

Plasma Exchange Complications are Related to the Venous Access RouteJeffrey Guptill

A Prospective, Randomized Controlled Trial of Valproic Acid in Ambulant Adults with SMA: The VALIANT TrialJohn Kissel

Amyloidosis and Exercise Tolerance in AN05 Muscular DystrophyMargherita Milone

MRI Quantification of Lower Limb Muscle Fatty Atrophy: A Potential Outcome Measure in Chronic NM DiseasesJasper Morrow

Identifying the Cause of Phenotypic Variability in a Family with NDMDips Raja Rayan

Sponsors

The meeting was sponsored by:

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

Annual Meeting Archive (2006-2010)

2010 MSG Annual Meeting

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

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

The meeting was sponsored by:

  • National Institutes of Health, 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

The Muscle Study Group received permission to post these presentations online:

Myositis Patient RegistryBob Goldberg

FOR-DMD Steroid TrialRobert Griggs

European Perspective on RecruitmentMichael Hanna

Update from British Myology SocietyMichael Hanna

Young Neurologists in the Basic LibraryAhmet Hoke

NIH Report on Support for Research on Neuromuscular DiseasesGlen Nuckolls

IMACS History and DevelopmentChester Oddis

Abstracts

The Muscle Study Group received permission to post the following abstracts online:

Safety and Feasibility of Transvenous Limb Profusion with Saline in Human Muscular DystrophyJames Howard

Mechanism of Axonal Degeneration in Drosophilia Model of Distal SBMAThomas Lloyd

Reproductability and Reliability of Quantitative MRI Measures for Neuromuscular DiseaseJasper Morrow

Clinical Improvement and Decreased Peripheral Autoreactivity in Refractory Myasthenia Gravis Treated with RituximabRichard Nowak

A Therapy for Duchenne Dystrophy Based Upon Inhibition of Mechanically Sensitive Ion ChannelsFrederick Sachs

Inherited Neuropathies ConsortiumCarly Siskind


2009 MSG Annual Meeting

The MSG held its annual meeting on September 21-23, 2009 at Beaver Hollow, Java Center, NY.

The 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.

The meeting was sponsored by:

  • National Institutes of Health:
    • 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
  • Santhera Pharmaceuticals

Presentations

The MSG received permission to post these presentations online:

The Peripheral Nerve Study Group – Its Rise and DemiseArthur Asbury

Treatment of Neuromuscular Diseases With Antisense OligonucleotidesFrank Bennett

FOR-DMDKate Bushby

MSG – Past, Present, FutureRobert Griggs

HYP-HOP Robert Griggs

The SMA ProjectJill Heemskerk

High Dose Vitamin C Treatment of CMT CMT-1ARichard Lewis

Dermal Nerve Assessment: A Window to the Mechanism of Neuropathies in Outcome MeasuresJun Li

The Challenges of Conducting Clinical Development in Rare/Orphan Diseases: The Industry PerspectiveThomas Meier

Attaining Symbiosis in Therapy Development Efforts in NMDsJohn Porter

Trial Design and Outcomes for Inherited Neuropathy NetworkDavide Pareyson

Abstracts

The MSG received permission to post the following abstracts online:

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

Using Video Gait Analysis to Assess Gait Dysfunction in Duchenne Muscular Dystrophy: A Pilot StudyYaacov Anziska

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

Mechanostat theory used to understand bone complications in children with Duchenne muscular dystrophyCraig Campbell

Sparing of Myofiber Membranes in Infantile Pompe’s DiseaseLudwig 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 studyThomas Meier

The challenges of conducting clinical development in rare/orphan diseases – The industry perspectiveThomas Meier

Diagnosis of Pompe Disease: Timing and Methods Used as Reported to the Pompe RegistryTim Miller

Fatty Acid Oxidation Disorders in Adults: A potentially treatable case of muscle diseaseRobert Pitceathly

Presence and Nature of “Tau” Immunoreactivity in Normal Myonuclei and Inclusion Body MyositisMohammad Salajegheh

Initial Results from Single Subcutaneous Administration of ACE-031, a Form of the Soluble Activin Type IIB Receptor, in Healthy Postmenopausal VolunteersJasbir 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 among 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.

The meeting was sponsored by:

  • National Institutes of Health:
    • 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

The MSG received approval to post the following presentations online:

Ocular MGMichael Benatar

Sleuthing Targets for Disease in MuscleAndrew Engel

Clinical Investigation of Neurological Channelopathies (CINCH) LandmarksRobert C. Griggs

HYP HOPRobert C. Griggs

Clinical Trials: From Inception to ImplementationLaura Herbelin

Trial Implementation StrategiesCornelia Kamp

Clinical Materials Services Unit (CMSU)Cornelia Kamp

Iplex Treatment of Myotonic DystrophyRichard T. Moxley III

High Dose Ascorbic Acid to Treat CMT1AMichael Shy


MSG 2007 Annual Meeting

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

The agenda 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.