2016 Muscle Study Group Scientific Annual Meeting

2016 Muscle Study Group Scientific Annual Meeting

Models of Neuromuscular Disease Across the Lifespan

September 24-26, 2016 in Snowbird, UT

Friday, September 23, 2016

We will have early check in from 6:00-7:00pm located at the top of the escalators for those who arrive early on Friday.

Day 1: Saturday, September 24, 2016

6:30am-7:40amBuffet Breakfast and Check-In

7:45am-5:00pmStudy Updates/Workshops

7:45am-8:30amMemantine Update-Richard J. Barohn, MD

8:30am-9:00amRasagiline Update- Richard J. Barohn, MD

9:00am-10:00amIPNEMA Update- Tahseen Mozaffar, MD

10:00am-11:00amArimoclomol Update- Mazen Dimachkie, MD

11:00am-12:00pmRRNMF- John Katz, MD

12:00am-1:30pmLunch

Muscle Study Group Executive Committee Meeting at this time

1:00pm-2:00pmMG-SCIg Update- Mazen Dimachkie, MD

2:00pm-3:00pmN103 BeatMG- Richard Nowak, MD

3:00pm-5:00pmYoung Investigator and UnderrepresentedNM Population Workshop

3:00pm-4:00pmPanel Discussion led byNicholas Johnson, MD and Chris Weihl, MD

4:00pm-5:00pmBreak Out Discussion Session

5:00pm-6:30pmDinner

6:45pmKeynote Speaker

Introduction by Chris Weihl, MD

Steve Greenberg, MD

Experiences in Academic Translational Medicine

8:00pmCampfire and S’mores Outdoor Reception

Day 2: Sunday, September 25, 2016

7:00am-8:00amBuffet Breakfast

2016 MSG Planning Committee Meeting

At this time we ask for people to set up their posters in Primrose for display

8:00am-8:30am Welcome and State of the Muscle Study Group Address

8:30am-10:10am Session I: DMD as a Model of Neuromuscular Disease Across the Lifespan

Moderator: Valeria Sansone, MD

8:35am-9:05amAnne Connolly, MD

Early Outcomes in DMD

9:10am-9:35amValeria Sansone, MD, PhD

Trial Design in DMD: Lessons Learned from Natural History

9:40am-10:05am Robert B. Weiss, PhD

Modifiers in DMD

10:10am-10:30amRefreshments Break

10:30am-12:05pmSession II: Extracellular Matrix Dysfunction as a Disease Mechanism

Moderator: Russ Butterfield, MD

10:40am-11:05amDean Burkin, PhD

The Myomatrix Reloaded: Integrin and ECM therapies for the muscular dystrophies

11:10am-11:35amKatherine Mathews, MD

Dystrogylcanopathy Cohort

11:40am-12:05pmCarsten Bonnemann, MD

Unknown

12:10pm-1:25pmLunch

CReATe Investigator Meeting at this Time

1:30pm-3:05pmSession III: Non-coding Repeats as a Disease Mechanism

Moderator: Chris Weihl, MD

1:40pm-2:05pmThomas A. Cooper, MD

Myotonic dystrophy as a paradigm for RNA gain function disease mechanisms

2:10pm-2:35pmCharles A. Thornton, MD

Update on myotonic dystrophy trial readiness

2:40pm-3:05pmNicholas Johnson, MD

Disease Progression and pathogenesis in congenital myotonic dystrophy

3:10pm-4:20pmWalk Through Poster Session

4:20pm-6:10pm Poster Session Presentations

Moderator: Valeria Sansone, MD

4:25pm-4:35pmW. David Arnold, MD

Open Label Trial of Ranolazine in Myotonia Congenita

4:40pm-4:50pmChad Heatwole, MD

PRISM-SMA: Patient Reported Impact of Symptoms in Adult Spinal Muscular Atrophy

4:55pm-5:05pmSummer Karafiath, MD

The Clinical Phenotype and Neuropathic Outcomes of Paclitaxel-Acute Pain Syndrome

5:10pm-5:20pmPedro Machado, MD

Baseline characteristics of a prospective natural history study of sporadic inclusion body myositis including MRI assessment

5:25pm-5:35pmMary Pautler, MPH

Limits of Postural Stability in Myotonic Dystrophy Compared to Normative Values

5:40pm-5:50pmMario Saporta, MD, PhD

Axonal RNA Profiling of Human Motor Neurons from Patients with Charcot-Marie-Tooth Disease as a Novel Approach to Study Axon Degeneration

5:55pm-6:05pmGita Ramdharry, PhD

Evaluating the benefits of community based aerobic training on the physical health and well-being of people with Neuromuscular disease

6:10pm-6:20pmDon McCorquodale, MD, PhD

RNA-Seq gene expression profiling identifies up-regulation of novel muscle specific gene musclin in adult and congenital myotonic dystrophy

6:30pm-7:30pmDinner

7:40pm Robert C. Griggs Annual MSG Keynote Speaker

Introduction by Richard J Barohn, MD

Francesco Muntoni, MD

Clinical Trials in DMD 2016: Where are we?

9:00pmYoung Investigator and Underrepresented NM Population Cocktail Reception

Day 3: Monday, September 26, 2016

7:00am-8:00amBreakfast

Workshop/Open dialogue Breakfast Seminar

for New Members and Guests

8:10am-9:55amSession IV: Mechanisms of Chaperone Dysfunction

Moderator: Nicholas Johnson, MD

8:10am-8:35amChris Weihl, MD

Chaperone Function and Dysfunction in Neuromuscular Disease

8:40am-9:05amRudolf Kley, MD

Proteomic Identification of Aggregates and Chaperones in Disease Muscle

9:10am-9:55amZohar Argov, MD

Phase 2 Trial Results of Autographic Manipulation with Trehalose in Oculopharyngeal Muscular Dystrophy

10:00am-10:30 amRefreshments Break

10:30am-1:05pmSession V: Autoimmunity

Moderator: Jeffrey Guptill, MD

10:40 am-11:05 amMark Anderson, MD

Mechanisms of Autoimmunity

11:10am-11:35amGil Wolfe, MD

MGTX: Results from the Thymectomy trial in non-thymomatous myasthenia gravis

11:40am-12:05amRenato Mantegazza, MD

Outcome Measures in Autoimmune NMJ diseases

12:10am-12:35pmJan L. Hillson, MD

Targeting FcRN in Neuromuscular Disease

12:40pm-1:05pmJon Lindstrom, PhD

AChR-Specific Immunosuppressive Therapy for MG

1:10pm-2:30pmLunch

PCORI Investigator Meeting at this time

2:40pm- 3:20pm Hot Topics Session

Osvaldo Nascimento, MD

Zika Virus Update saliva

James Howard, MD

REGAIN: A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy if Eculizumab in Subjects with Refractory Generalized Myasthenia Gravis

3:20pm-4:40pm Session VI: Mentoring for Young Investigators and Underrepresented NM Population to Promote IncreasedEngagement in Neuromuscular Research

Moderator: Michael Benatar, MD

3:30pm-3:50pm Glen Nuckolls, PhD

NIH Programs for Young Investigators

3:55pm-4:15pm Richard J. Barohn, MD

Advice for Young Investigators

4:20pm-4:40pm Jeffrey Guptill, MD

Research Support Resources for Junior Investigators

4:50pm-6:50pm Break/ Other Muscle Study Group Committee Meetings

7:00pm-8:15pm Dinner