PDF MONDAY, SEPTEMBER 17, 2018

MUSCLE STUDY GROUP ANNUAL SCIENTIFIC MEETING

SUNDAY, SEPTEMBER 16, 2018

2018

We will have early check in from 4-7 p.m. located in the Porter's Lodge.

DAY 1: MONDAY, SEPTEMBER 17, 2018

7:30-8 a.m.

8-9 a.m. 9-9:30 a.m.

9:30 a.m.-12 p.m. 9:30-9:45 a.m. 9:45 - 10 a.m. 10-10:15 a.m. 10:15-10:30 a.m. 10:30-10:45 a.m.

10:45-11 a.m. 11:-11:15 a.m. 11:15-11:30 a.m.

Check-In Bernard Sunley Building Foyer

We ask for posters to be set up in Room C for display before 6 p.m.

Breakfast Dining Hall

Welcome and State of the Muscle Study Group Address Lecture Hall Richard J. Barohn, M.D., and Prof Michael G. Hanna

Update to MSG Meetings Lecture Hall

Opening Remarks Richard J. Barohn, M.D.

NIH sIBM Nautral History Study Tahseen Mozaffar, M.D.

Memantine Richard J. Barohn, M.D.

IPANEMA Tahseen Mozaffar, M.D.

IBM International Genetics Consortium Study Pedro Machado, M.D., and Prof Michael G. Hanna

MGSCIg Mazen Dimachkie, M.D.

Arimoclomol in IBM Mazen Dimachkie, M.D.

FOR-DMD Robert Griggs, M.D.

11:30-11:45 a.m. 12-12:30 p.m. 12:30-1:30 p.m. 1:30-3 p.m.

1:30-2 p.m.

A Randomized, Prospective Trial of Immunosuppressant Dose Reduction in Stable Myasthenia Gravis Michael Hehir, M.D.

Muscle Study Group Executive Committee Meeting Room B

Lunch Dining Hall Sponsor: Sarepta

Young Investigator Support Session Lecture Hall Expert Panel: Michael McDermott, Ph.D. (Statistics), Bill Martens (Database), Dr. David Bennett and Prof Michael G. Hanna

1st Presentation

2-2:30 p.m. 2:30-3 p.m.

2nd Presentation 3rd Presentation

3-5 p.m. 3:10-3:30 p.m.

Optional Walking Tour (16 spots available, first come first served) Registration Desk

Break

3:30-6 p.m. 6:15-7:30 p.m. 7:30-9 p.m.

FOR DMD Meeting Lecture Hall

Dinner Dining Hall

Walk Through Poster Session & Evening Reception Room C

DAY 2: TUESDAY, SEPTEMBER 18, 2018

8-9 a.m.

Breakfast Dining Hall Sponsor: MT Pharma

9-10:35 a.m. 9-9:20 a.m. 9:25-9:45 a.m.

9:50-10:10 a.m. 10:15-10:35 a.m.

Session I: New technologies supporting rehabilitation in NMDs Lecture Hall Moderator: Jeffrey Statland, M.D.

Use of online and web-based support for people with neurological conditions Prof Lorna Paul, Glasgow, UK

Advances in 3D printing to develop custom made orthoses for people with NMDs Elizabeth Wojciechowski, MSc, BEng, Sydney, Australia

Integrating apparel and robotics to improve mobility in persons with neuromuscular disease Tina Duong, MPT, Ph.D., Stanford, CA

Participation in gaming: co-production of solutions with patients, charities, industry and academia Dr. Gita Ramdharry, London, UK

10:40-11 a.m.

Break

11 a.m.-1 p.m.

11-11:20 a.m. 11:25-11:45 a.m. 11:50-12:10 p.m.

12:15 -12:35 pm 12:40-1:00 pm

Session II: The future of neuromuscular disease is here: implications for clinical practice Lecture Hall Moderator: Aisling Carr, M.D.

Practical implications of the introduction of new gene therapies for Transthyretin amyloidosis Prof Julian Gillmore, London, UK

Neuromuscular MRI: current and future clinical practice Dr. Jasper Morrow, London, UK

The changing natural history in Duchennes muscular dystrophy, supporting the transition into independent adult life Dr. Ros Quinlivan, London, UK

New molecular therapies in congenital myasthenic syndromes Prof David Beeson, Oxford, UK

The WalkWithPath work with NM diseases Lise Pape, Human Biology BSc, London, UK

1-2 p.m.

Lunch Dining Hall Sponsored by AveXis

2-3 p.m.

3-3:50 p.m. 3-3:10 p.m.

3:10-3:20 p.m. 3:20-3:30 p.m. 3:30-3:40

3:40-3:50 4-4:25 p.m. 4:30-5:55 p.m. 4:30-4:50 p.m. 5:00-5:20 pm 5:30-5:50 pm 5:55-6:15 pm 7:30-9 p.m.

Robert C. Griggs Annual Muscle Study Group Keynote Speaker Lecture Hall

A Small Circle of Life; genetics, disease and prevention Professor Sir Doug Turnbull

Platform Presentation: Session I Lecture Hall Moderators: Pedro Machado, M.D., and Jeffrey Statland, M.D.

The genomics of arthrogryposis: novel candidate genes, evidence for oligogenic inheritance and the importance of reanalysis Davut Pehlivan, Ph.D.

Targeting the heat shock response in sporadic IBM patient-derived fibroblasts Charlotte Spicer, Ph.D.

First-in-human study of AT-GAA (ATB200/AT2221) in patients with Pompe disease: preliminary results from the ATB200-02 Trial Tahseen Mozaffar, M.D.

The "hidden" genome: a novel bioinformatic approach for extracting and analysing mitochondrial DNA from nuclear genomic high throughput sequence data at UCL Queen Square Institute of Neurology Olivia Poole, MBBS, MRCP

Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9 Guangbin Xia, M.D., Ph.D.

Group Photo Location to be determined

Session III: New technology supporting clinical trials Lecture Hall Moderator: Lindsay Alfano, DPT

Activity monitoring in NMDs: successes, challenges, and path forward Linda Lowes, PT, Ph.D., Columbus, OH

Use of decision support tools to inform families and support shared decision making for pediatric neuromuscular trials Holly Peay, Ph.D., MS, Research Triangle Park, NC

Novel observer-reported outcome assessment for use in clinical trials in DMD Mindy Leffler, MEd, Seattle, WA

Latest genetic therapy developments in nm diseases Prof Matthew Wood, Oxford, UK

Gala Dinner Dining Room Business Dress Sponsor: MT Pharma

DAY 3: WEDNESDAY, SEPTEMBER 19, 2018

8-9 a.m. 9-9:50 am 9-9:10 a.m. 9:10-9:20 a.m.

9:20-9:30 a.m. 9:30-9:40 a.m.

9:40-9:50 a.m.

Breakfast Dining Hall

Platform Presentation: Session II Lecture Hall Moderator: Pedro Machado, M.D.

Inflammation Inhibits Muscle Regeneration in a Xenograft Model of Sporadic Inclusion Body Myositis Thomas Lloyd, M.D., Ph.D.

Clinical Outcome Study for Dysferlinopathy: Three years of natural history data for clinical trial readiness Meredith James, BSc

Poor muscle endurance of swallowing muscles in myositis patients with dysphagia Tae Chung, M.D.

Mexiletine for Myotonic Dystrophy Type 1 (DM1): Results from a Randomized, Placebo Controlled, Clinical Efficacy Trial Chad Heatwole, M.D.

The RePOWER Study: A Global Prospective Observational Study of Patients with Primary Mitochondrial Myopathy John Vissing, M.D., Ph.D.

9:50-10:15 a.m. 10:15-11:35 a.m. 10:15-10:40 a.m. 10:45-11:05 a.m. 11:10-11:35 a.m. 12:30-1:30 pm

Break

Check out of room if departing today

Session IV: Future Treatment Lecture Hall Moderator: Jeffrey Statland, M.D.

Ethics of Human Gene Editing Robin Lovell-Badge, Ph.D, Francis Crick Institute

The benefits of RNA analysis for muscle disease diagnosis Beryl Cummings, BSc, Broad Institute

Brain Computer Interfaces Prof Peter Desain, Donders Institute

Lunch Dining Hall Sponsored by PTC Pharma

1:30-3:00 pm

1:30 -1:50 pm 1:55-2:15 pm 2:20-2:40 pm 2:45-3:05 pm

Session V: Sponsor Trials/Industry Late Breaking News Lecture Hall Moderator: Prof Michael Hanna

PTC

AveXis

MT Pharma

Sarepta

3:05

Close Out Prof Michael Hanna

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