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Agenda

2020 MDA Clinical and Scientific Conference

Previous Sessions

Date Time Session Name
June 18 3:00 PM Virtual Industry Forum: Presented by Sanofi Genzyme
Respiratory Involvement in Neuromuscular Disorders: Pompe Disease as a Prototype
Namita Goyal, MD
Michelle Cao, DO
June 19 4:00 PM Small Molecules & Therapeutic Antibodies: Experimental, Preclinical, Clinical (Now Available On-Demand)

Stathmin-2 loss a new hallmark forTDP-43-dependent Amyotrophic Lateral Sclerosis
Jone Lopez-Erauskin, PhD (Chair)

VCP inhibitor CB-5083: a potential treatment for VCP inclusion Body Myopathy
Virginia Kimonis, MD, MRCP (Chair)

Inhibition of PKC alleviates nucleocytoplasmic transport disruption in FUS-mediated ALS
Clotilde Lagier-Tourenne, MD, PhD

“Designer DNA drug” therapies for sporadic ALS: restoration of stathmin-2oruse of “identity theft” to generate replacement neurons
Don Cleveland, PhD

Update on Rare Disease Programs at Denali
Joseph Lewcock, PhD

July 10 4:00 PM Implications of NBS in Clinical Practice - What Does the Future Hold? (CME Certified) (Now Available On-Demand)

Session Moderator
R. Rodney Howell, MD (Chair)

Newborn Screening for Neuromuscular Conditions
Brittany Hernandez

Newborn Screening: How to Prepare Your MDA Care Center
Marcia Felker, MD

Newborn Screening for SMA: Wisconsin Experience
Mei Baker, MD

July 17 4:00 PM When Gut Feelings Aren’t Helping - Navigating Complicated Ethics in the Neuromuscular Clinic (CME Certified) (Now Available On-Demand)
John Brandsema, MD (Chair)
Alyssa Burgart, MD, MA
Julie Parsons, MD
Thomas Crawford, MD
July 27 5:45 PM Virtual Industry Forum: Presented by PTC (Now Available On-Demand)
Duchenne Muscular Dystrophy: Examining the Totality of Evidence for Corticosteroid Treatment
Vamshi Rao, MD (Chair)
Perry Shieh, MD, PhD
Armando Villalta, PhD
July 28 4:00 PM Virtual Industry Forum: Presented by Biogen
Time to SHINE on SMA: The Nusinersen Clinical Development Program Including Long-Term Outcomes in Infants and Children
Diana Castro, MD
Thomas Crawford, MD
July 31 4:00 PM Ultra-Rare Myopathies and Dystrophies (Now Available On-Demand)
Alan Beggs, PhD (Chair)
Angela Lek, PhD (Chair)
Frances Evesson, PhD
Scot Wolfe, PhD
Asli Aykanat, MD
August 6 3:00 PM Virtual Industry Forum: Presented by Biogen
Later-onset SMA: Perception of Stability and a Treatment for a Neuromuscular Disease
Crystal Proud, MD
Molly Stark, DPT
Kathleen McMahon, RN
Patient Ambassador Dallas F.
August 7 4:00 PM Clinical Trial Design (CME Certified) (Now Available On-Demand)

Session Moderator
Merit Cudkowicz, MD (Chair)

Session Moderator
Richard Finkel, MD (Chair)

Biostatistical considerations in clinical trial design in neuromuscular disorders
Ken Cheung, PhD

Clinical considerations for efficient design of clinical trials for neuromuscular diseases
Kevin Flanigan, MD

“Common” neuromuscular diseases, with large-scale studies, using ALS as an example
Sabrina Paganoni, MD

Rare neuromuscular disease considerations, using giant axonal neuropathy and myotubular myopathy as examples
Diana Bharucha-Goebel, MD

August 12 2:00 PM Virtual Industry Forum: Presented by Sarepta Therapeutics
Evolving Diagnostic Pathway & Gene Therapies in Development for the Limb-Girdle Muscular Dystrophies
Matthew Wicklund, MD
Eric Pozsgai, PhD
Louise Rodino-Klapac, PhD
August 14 4:00 PM Lab to Life: What's New in ALS & CMT (Now Available On-Demand)
Matthew Harms, MD (Chair)
Michael Shy, MD (Chair)
Stephan Zuchner, PhD
August 19 4:00 PM Best Practices in Communication in NMD (This program is offered for up to .15 (or 90 minutes) ASHA CEU's, Intermediate level, professional area)
Angela Strauch Lane, M.S., CCC-SLP
Kelly Casey, OTD, OTR/L, BCPR, ATP, CPAM
August 20 12:00 PM Virtual Industry Forum: Presented by Alexion Pharmaceuticals
The Role of Complement in Neuromuscular Disorders
Ahmed Enayetallah, MD PhD
August 28 4:00 PM Real World Data: Disease Registries, Natural History & Post-Marketing Surveillance (CME Certified) (Now Available On-Demand)
Paul Mehta, MD (Chair)
Paula Clemens, MD (Chair)
Craig Campbell, MD
Craig McDonald, MD
Jane Larkindale, DPhil
Jeff Rothstein, MD, PhD
August 31 4:00 PM Best Practices in Swallowing in ALS (This program is offered for up to 0.1 (or 60 minutes) of ASHA CEU's, Intermediate level, professional area)
Donna C. Tippett, MPH, MA CCC-SLP
September 11 2:00 PM Preclinical Trial Design and Biomarker Development (Now Available On-Demand)
Kanneboyina Nagaraju, DVM, PhD (Chair)
Jim Dowling, MD (Chair)
Laurent Bogdanik, PhD
Yetrib Hathout, PhD
Yi-Wen  Chen, Ph.D., M.S., D.V.M
September 14 2:00 PM

Virtual Industry Forum: Presented by Sarepta Therapeutics
Natural History and Therapeutic Options in Development for DMD
Craig McDonald, MD
Crystal Proud, MD
Louise Rodino-Klapac, PhD

Jahannaz Dastgir, DO

September 18 3:00 PM Lab to Life: What's New in LGMD, FSHD & DM (CME Certified)
Charles Thornton, MD (Chair)
Katherine Mathews, MD (Chair)
Tom Cooper, MD
Laura Ranum, PhD
Katelyn Daman
Steve Tapscott, MD, PhD
Matthew Wicklund, MD
Melissa Spencer, PhD
September 21 3:00 PM Virtual Industry Forum: Presented by Genentech
Expanding Possibilities in the Treatment of Spinal Muscular Atrophy: Introducing Evrysdi™ (risdiplam)
Perry Shieh, MD, PhD
September 29 2:00 PM Virtual Industry Forum: Presented by AveXis
Gene therapy in the Clinic: Evolution and Clinical Considerations
Diana Bharucha-Goebel
Arthur Burghes
Petra Kaufmann

Once your registration is completed, a link will be provided to access our Summer Seminar Series agenda and RSVP information, Virtual Poster Library and on-demand presentations. If you have already registered for the 2020 MDA Clinical & Scientific Conference, please use this link to access these offerings and archived content. Your RSVP is required to gain access to our upcoming programs.