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)
Amy Pasternak, PT, DPT, PCS
Erin Sim, OT
Tim Estilow, OT
Julie Wells, OT
Claudia Senesac, PT
Asia 1
Workshop
CME Certified
Best Practices in Communication & Swallowing in NMD
Angela Strauch Lane, MS, CCC-SLP (Chair)
Donna Tippett, MPH, MA, CCC-SLP (Chair)
Kelly Casey, OTD, OTR/L, BCPR, ATP, CPAM
Maureen A. Lefton-Greif, PhD, CCC-SLP, BCS-S, ASHA Fellow
Lenie van den Engel-Hoek, PhD
Janine (Genevieve) McKeon, MS, CCC-SLP
Asia 3
Workshop
CME Certified
Respiratory Manifestations of Neuromuscular Diseases
Barbara Smith, PT, PhD (Chair)
John Hansen-Flaschen, MD (Chair)
Hank Mayer, MD
Howard Panitch, MD
Phil Choi, PhD
Amen Sergew, MD
Jason T. Ackrivo, MD, MSCE
Jenny Cheng, MD
Asia 4
Workshop
CME Certified
Mental Health Considerations in Neuro Muscular Disease
Sarah Stoney, MSW, LSW (Chair)
Diane Murrell, LCSW (Chair)
James M. Denham, MDiv, BCC
Vanessa Battista, MS, RN, CPNP
Asia 5
3 PM – 6 PM MDA Scholars Networking Session & Reception (Invitation Only)
6 PM – 8 PM Welcome & Poster Reception in Exhibit Hall | Atlantic

MONDAY, MARCH 23, 2020

6:45 AM – 7:45 AM
Industry Forum Breakfast Presented by Biogen
Pacific A
Industry Forum Breakfast Presented by Sanofi Genzyme
Northern Hemisphere Salon B, C
8:30 AM – 10 AM Opening Session with Keynote Panel Discussion | Pacific B
Exhibit Hall Open 10 AM – 8:30 PM
10 AM – 10:30 AM Break in Exhibit Hall | Atlantic
10:30 AM – NOON
Practical Considerations in Manufacture & Use of Gene-Targeted Therapies
Dongsheng Duan, PhD (Chair)
Kevin Flanigan, MD (Chair)
Dirk Grimm, PhD
David Favre, PhD
Northern Hemisphere Salon D
Muscle Regeneration in Disease
Matthew Alexander, PhD (Chair)
Emanuela Gussoni, PhD (Chair)
Michael Rudnicki, OC, PhD, FRSC
Justin Boyer, PhD
April Pyle, PhD
Nenad Bursac, PhD
Southern Hemisphere I, II
CME Certified Meeting the Unaddressed Behavioral & Mental Health Needs of NMD Patients
Sarah Stoney, MSW, LSW (Chair)
Laurie Gutmann, MD (Chair)
Erin Springer, RN
Southern Hemisphere III, IV, V
NOON – 1:15 PM
Industry Forum Lunch Presented by AveXis
Northern Hemisphere Salon B, C
Industry Forum Lunch Presented by PTC Therapeutics
Northern Hemisphere Salon E
Industry Forum Lunch Presented by Sarepta Therapeutics
Pacific A
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)
Stephan Zuchner, MD, PhD
Michael Benatar, MBChB, MS, DPhil, FAAN, FANA
Northern Hemisphere Salon D
Preclinical Trial Design & Biomarker Development
Kanneboyina Nagaraju, DVM, PhD (Chair)
James Dowling, MD, PhD (Chair)
Laurent Bogdanik, PhD
Yi-Wen Chen, PhD, MS, DVM
Yetrib Hathout, PhD
Southern Hemisphere I, II
CME Certified Implications of NBS in Clinical Practice – What Does the Future Hold?
R. Rodney Howell, MD (Chair)
Marcia Felker, MD
Natasha Bonhomme
Mei Baker, MD
Brittany Hernandez
Southern Hemisphere III, IV, V
3 PM – 3:30 PM Break in Hemispheres Foyer
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)
Matthew Wicklund, MD
Melissa Spencer, PhD
Katelyn Daman, PhD
Stephen Tapscott, MD, PhD
Tom Cooper, MD
Laura Ranum, PhD
Northern Hemisphere Salon D
Ultra-Rare Myopathies and Dystrophies
Alan Beggs, PhD (Chair)
Angela Lek, PhD (Chair)
Scot Wolfe, PhD
Frances Evesson, PhD
Southern Hemisphere I, II
Trainee Presentations from Abstracts
Elizabeth McNally, MD, PhD (Chair)
Monkol Lek, PhD (Chair)
Southern Hemisphere I, II
CME Certified Real World Data: Disease Registries, Natural History & Post-Marketing Surveillance
Paula Clemens, MD (Chair)
Paul Mehta, MD (Chair)
Craig McDonald, MD
Craig Campbell, MD
Jane Larkindale, DPhil
Jeff Rothstein, MD, PhD
Southern Hemisphere III, IV, V
6 PM – 8 PM Networking & Poster Reception in Exhibit Hall | Atlantic

Tuesday, MARCH 24, 2020

5:30 AM – 6:30 AM MDA Team Momentum Fun Run & Mini MDA Muscle Walk
Causeway between Dolphin and Swan Hotel
6:45 AM – 7:45 AM
Industry Forum Breakfast Presented by Dyne Therapeutics
Pacific A
8 AM – 10 AM
Neuromuscular Junction in Disease
Hanns Lochmüller, MD, PhD, FAAN (Chair)
Mark Rich, MD, PhD (Chair)
William Arnold, MD
Marcus Ruegg
Erik Storkebaum, PhD
Southern Hemisphere I
Effect of Neuromuscular Diseases in Other Tissues
Alyson Fiorillo, PhD (Chair)
Elizabeth McNally, MD, PhD (Chair)
Michael Huang, PhD
Miranda Grounds, PhD
Stephanie Duguez, PhD
Maury Swanson, MS, PhD
Southern Hemisphere II
CME Certified The Future of Neuromuscular Care
Barry Byrne, MD, PhD (Chair)
John Day, MD, PhD (Chair)
Susan Apkon, MD
Russell Butterfield, MD, PhD
Matthew Wicklund, MD
Sharon Hesterlee, PhD
Southern Hemisphere IV, V
CME Certified Genetic Testing: VUS Path Correlation, Future of Genetic Testing, NBS
Jennifer Roggenbuck, MS, LGC (Chair)
Kathryn Wagner, MD, PhD (Chair)
Monkol Lek, PhD
Julie Cohen, ScM
Robert Nussbaum, MD
Carolina Tesi-Rocha, MD
Southern Hemisphere IIIstrong>
Exhibit Hall Open 10 AM – 8:30 PM
10 AM – 10:30 AM Break in Hemispheres Foyer
10:30 AM – NOON
Small Molecules & Therapeutic Antibodies: Experimental, Preclinical, Clinical
Jone López-Erauskin, PhD (Chair)
Virginia Kimonis, MD, MRCP (Chair)
Don W. Cleveland, PhD
Clotilde Lagier-Tourenne, MD, PhD
Joseph Lewcock, PhD
Southern Hemisphere I
Single Cell Analysis Approaches
Eric Wang, PhD (Chair)
Melissa Spencer, PhD (Chair)
Haibin Xi, PhD
Silvere van der Maarel, PhD
Charlotte Peterson, PhD
Armando Villalta, PhD
Southern Hemisphere II
CME Certified Adult Care of Pediatric Onset Diseases & Transitions in Care
Anne Connolly, MD (Chair)
Dennis Matthews, MD (Chair)
Jaime Twanow, MD
Arun Varadhachary, MD, PhD
Southern Hemisphere IV, V
CME Certified Clinical Trial Design
Merit Cudkowicz, MD (Chair)
Richard Finkel, MD (Chair)
Sabrina Paganoni, MD
Diana Bharucha-Goebel, MD
Kevin Flanigan, MD
John Day, MD, PhD
Southern Hemisphere III
NOON – 1:15 PM
Industry Forum Lunch Presented by Biogen
Pacific A
Industry Forum Lunch Presented by Genentech
Northern Hemisphere Salon B, C
Industry Forum Lunch Presented by Sarepta Therapeutics
Northern Hemisphere Salon E
1:15 PM – 3 PM
CME Certified Clinical Trial Presentations Part 1
Stanley Appel, MD (Chair)
Han Phan, PhD
Pacific B
3 PM – 3:30 PM Break in Exhibit Hall | Atlantic
3:30 PM – 6:30 PM
CME Certified Clinical Trial Presentations Part 2
George Nomikos, MD
Francesco Muntoni, MD
Ines Macias-Perez, MD
Michelle Mellion, MD
Erica Finanger, MD
Gary Pattee, MD
James Howard, MD
Pacific B
6:30 PM – 8:30 PM Networking & Poster Reception in Exhibit Hall | Atlantic

WEDNESDAY, MARCH 25, 2020

8 AM – 10 AM
CME Certified Gene-Targeted Therapy
Matthew Harmelink, MD (Chair)
Diana Castro, MD (Chair)
John Day, MD, PhD
Anne Connolly, MD
Perry Shieh, MD, PhD
Michael Lawlor, MD, Phd
Robert Moravec, MD
Pacific B
10 AM – 10:30 AM Break in Pacific 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)
Julie Parsons, MD
Thomas Crawford, MD
Alyssa Burgart, MD
Pacific B
12:30 PM MEETING ADJOURNED