Agenda

Agenda

Saturday, March 21, 2020

5 PM - 8 PM MDA Care Center Directors Reception & Dinner (Invitation Only)

SUNDAY, MARCH 22, 2020

7:30 AM – 3 PM MDA Care Center Directors Meeting (Invitation Only)
1:15 PM – 6 PM
Workshop
CME Certified
PT & OT Best Practices: Updates in ALS, CMT, DMD, SMA
Gabrielle Steinhorn, PT, DPT, NCS (Chair)
Allan Glanzman, PT, DPT, PCS (Chair)
Workshop
CME Certified
Best Practices in Communication & Swallowing in NMD
Angela Strauch Lane, MS, CCC-SLP (Chair)
Donna Tippett, MPH, MA, CCC-SLP (Chair)
Workshop
CME Certified
Respiratory Manifestations of Neuromuscular Diseases
Barbara Smith, PT, PhD (Chair)
John Hansen-Flaschen, MD (Chair)
Workshop Mental Health Considerations in Neuro Muscular Disease
Sarah Stoney, MSW, LSW (Chair)
Diane Murrell, LCSW (Chair)
3 PM – 6 PM MDA Scholars Networking Session & Reception (Invitation Only)
6 PM – 8 PM Welcome & Poster Reception in Exhibit Hall

MONDAY, MARCH 23, 2020

6:45 AM – 7:45 AM
Industry Forum Breakfast Presented by Biogen
Industry Forum Breakfast Presented by Sanofi Genzyme
8:30 AM – 10 AM Opening Session with Keynote Panel Discussion
Exhibit Hall Open 10 AM – 8:30 PM
10 AM – 10:30 AM Break in Exhibit Hall
10:30 AM – NOON
Practical Considerations in Manufacture & Use of Gene-Targeted Therapies
Dongsheng Duan, PhD (Chair)
Kevin Flanigan, MD (Chair)
Muscle Regeneration in Disease
Matthew Alexander, PhD (Chair)
Emanuela Gussoni, PhD (Chair)
CME Certified Meeting the Unaddressed Behavioral & Mental Health Needs of NMD Patients
Sarah Stoney, MSW, LSW (Chair)
Laurie Gutmann, MD (Chair)
NOON – 1:15 PM
Industry Forum Lunch Presented by AveXis
Industry Forum Lunch Presented by PTC Therapeutics
Industry Forum Lunch Presented by Sarepta Therapeutics
1:15 PM – 3 PM
CME Certified Lab to Life: What's New in ALS & CMT, Molecular Pathways, Mutations, Phenotype-Genotype Correlations
Matthew Harms, MD (Chair)
Robert Baloh, PhD (Chair)
Michael Shy, MD (Chair)
Preclinical Trial Design & Biomarker Development
Kanneboyina Nagaraju, DVM, PhD (Chair)
James Dowling, MD, PhD (Chair)
CME Certified Implications of NBS in Clinical Practice – What Does the Future Hold?
R. Rodney Howell, MD (Chair)
3 PM – 3:30 PM Break in Exhibit Hall
3:30 PM – 6 PM
CME Certified Lab to Life: What's New in DM, LGMD, FSHD (Autosomal Dominant Subtypes): Molecular Pathways, Mutations, Phenotype-Genotype Correlations
Katherine Mathews, MD (Chair)
Charles Thornton, MD (Chair)
Ultra-Rare Myopathies and Dystrophies
Alan Beggs, PhD (Chair)
Angela Lek, PhD (Chair)
Trainee Presentations from Abstracts
Elizabeth McNally, MD, PhD (Chair)
Monkol Lek, PhD (Chair)
CME Certified Real World Data: Disease Registries, Natural History & Post-Marketing Surveillance
Paula Clemens, MD (Chair)
Paul Mehta, MD (Chair)
6 PM – 8 PM Networking & Poster Reception in Exhibit Hall

Tuesday, MARCH 24, 2020

5:30 AM – 6:30 AM Fun Run & Mini MDA Muscle Walk
6:45 AM – 7:45 AM
Industry Forum Breakfast Presented by Dyne
8 AM – 10 AM
Neuromuscular Junction in Disease
Hanns Lochmüller, MD, PhD, FAAN (Chair)
Mark Rich, MD, PhD (Chair)
Effect of Neuromuscular Diseases in Other Tissues
Alyson Fiorillo, PhD (Chair)
Elizabeth McNally, MD, PhD (Chair)
CME Certified The Future of Neuromuscular Care
Barry Byrne, MD, PhD (Chair)
John Day, MD, PhD (Chair)
CME Certified Genetic Testing: VUS Path Correlation, Future of Genetic Testing, NBS
Jennifer Roggenbuck, MS, LGC (Chair)
Kathryn Wagner, MD, PhD (Chair)
Exhibit Hall Open 10 AM – 8:30 PM
10 AM – 10:30 AM Break in Exhibit Hall
10:30 AM – NOON
Small Molecules & Therapeutic Antibodies: Experimental, Preclinical, Clinical
Jone López-Erauskin, PhD (Chair)
Virginia Kimonis, MD, MRCP (Chair)
Single Cell Analysis Approaches
Eric Wang, PhD (Chair)
Melissa Spencer, PhD (Chair)
CME Certified Adult Care of Pediatric Onset Diseases & Transitions in Care
Anne Connolly, MD (Chair)
Dennis Matthews, MD (Chair)
CME Certified Clinical Trial Design
Merit Cudkowicz, MD (Chair)
Richard Finkel, MD (Chair)
NOON – 1:15 PM
Industry Forum Lunch Presented by Biogen
Industry Forum Lunch Presented by Genentech
Industry Forum Lunch Presented by Sarepta Therapeutics
1:15 PM – 3 PM
Clinical Trial Presentations Part 1
Sharon Hesterlee, PhD (Chair)
Stanley Appel, MD (Chair)
3 PM – 3:30 PM Break in Exhibit Hall
3:30 PM – 6:30 PM
Clinical Trial Presentations Part 2
Sharon Hesterlee, PhD (Chair)
Stanley Appel, MD (Chair)
6:30 PM – 8:30 PM Networking & Poster Reception in Exhibit Hall

WEDNESDAY, MARCH 25, 2020

8 AM – 10 AM
CME Certified Gene-Targeted Therapy
Matthew Harmelink, MD (Chair)
Diana Castro, MD (Chair)
10 AM – 10:30 AM Break in Hemispheres Foyer
10:30 AM – 12:30 PM
CME Certified When Gut Feelings Aren’t Helping – Navigating Complicated Ethics in the Neuromuscular Clinic
John Brandsema, MD (Chair)
12:30 PM MEETING ADJOURNED