2021 MDA Virtual Clinical & Scientific Conference

March 15 – 18, 2021

The 2021 MDA Virtual Clinical & Scientific Conference will take place March 15-18, 2021, in an all-virtual environment, which will include scheduled live-broadcast sessions, on-demand videos, virtual networking sessions, exhibits and poster sessions.

This conference platform is supported by an independent education grant from Pfizer.

6 AM – 6 PM ET

Poster Sessions Open

9:30 – 10 AM ET

Welcome & Keynote Presentation

10 AM – 6 PM ET

Exhibit Hall Open

10 – 11 AM ET

New Biomarkers in Neuromuscular Disease Research

Elizabeth McNally
MD, PhD (Chair)
Annemieke Aartsma-Rus
PhD
Glenn Walter
PhD
Thurman Wheeler
MD
11 AM – 12 PM ET

MOVR & Real World Evidence

Rayne Rodgers
MPH (Chair)
Susan Apkon
MD
DeWayne Townsend
DVM, PhD
12 – 1:30 PM ET

Industry Forum Lunch Presented by Alexion Pharmaceuticals

A Treatment for Adult Patients With Anti-Acetylcholine Receptor (AChR) AntibodyPositive Generalized Myasthenia Gravis (gMG)

Industry Forum Lunch Presented by Genentech

Evrysdi® (risdiplam) Experiences: Explore the Science and Hear From People Living With Spinal Muscular Atrophy

Industry Forum Lunch Presented by Novartis Gene Therapies

Adena-Associated Virus (AAV) Platform: Considerations for Gene Therapy

1:30 – 3 PM ET

Axonal Degeneration and Transport Defects

Michael Shy
MD (Chair)
Robert Baloh
MD
Michael Coleman
PhD
Erika Holzabaur
PhD
Charlotte Sumner
MD

Therapeutic Considerations for Dominant Neuromuscular Diseases

Beth Barton
PhD (Chair)
Veronique Bolduc
PhD
Andrew Findlay
MD
Eugene Yeo
PhD, MBA
Toshifumi Yokota
PhD
Romesh Subramanian
PhD
3 – 4:30 PM ET

Gene Editing in Neuromuscular Disease

Eric Olson
PhD (Chair)
Charles Gersbach
PhD
Erik Sontheimer
PhD

Innovative Modeling in Neuromuscular Disease

Jamshid Arjomand
PhD (Chair)
Robert Bloch
PhD
Clive Svendsen
PhD
Mina Gouti
PhD
Eleonora Maino
M.Sc
4:30 – 6 PM ET

Non-viral Delivery in Neuromuscular Disease

Stanley C. Froehner
PhD (Chair)
Oxana Beskrovnaya
PhD
James Dahlman
PhD
Melissa Spencer
PhD
Nicholas Whitehead
PhD
6 – 8 PM ET

MDA Scholars Virtual Meeting (Invitation Only)

6 AM – 9 PM ET

Exhibit & Poster Hall Open

8 – 9 AM ET

Keynote & Welcome Presentation

9 – 10 AM ET

Lab-to-Life: Inflammatory Myopathies

Thomas Lloyd
MD, PhD (Chair)
Steven Greenberg
MD
Andrew Mammen
MD, PhD
Lauren Pachman
MD
10 – 11 AM ET

Lab-to-Life: Mitochondrial Diseases including Friedreich's Ataxia

Carlos Moraes
PhD (Chair)
Gino Cortopassi
PhD
Anu Soumalainen
MD, PhD
11 AM – 12 PM ET

Lab-to-Life: Spinal and Bulbar Muscular Atrophy

Kenneth (Kurt) Fischbeck
MD (Chair)
Andrew Lieberman
MD, PhD
Diane Merry
PhD
Carlo Rinaldi
MD, PhD
12 – 1:30 PM ET

Industry Forum Lunch Presented by Amylyx Pharmaceuticals

Pathways and Paradigms in ALS: Life and Death in the Neuron

Industry Forum Lunch Presented by Biogen

The Evolution of Treatment with SPINRAZA® (nusinersen) Over the Last4 Years

Industry Forum Lunch Presented by Sarepta Therapeutics

Understanding Diagnosis and Phenotypes in Muscular Dystrophies

1:30 – 2:30 PM ET

Lab-to-Life: Congenital MD

Kevin Campbell
PhD (Chair)
Carsten Bonnemann
MD
Madeline Durbeej-Hjalt
PhD
Qi Lu
MD, PhD
2:30 – 4 PM ET

Barriers to Clinical Trial Participation (CME Accredited Session)

John Day
MD, PhD (Chair)
Kate Eichinger
PT, PhD, NCS
Colleen Labbadia
Parent
Catherine Seiner
PT, MHS

Optimizing ALS Clinical Care and Clinical Research in an MDA ALS Care Center in 2021 (CME Accredited Session)

Lora Clawson
MSN, CRNP(Chair)
Kristen Riley
PhD
Rachel Slusher
PT, DPT
Alpa Uchil
MSN, MPH, CRNP

The Changing Regulatory Environment: Impact on Neuromuscular Disease Drug Development

James Valentine
(Chair)
Teresa Buracchio
MD
Michelle Campbell
PhD
Jeff Castelli
PhD
Mindy Leffler
Paul Melmeyer
Lei Xu
MD, PhD
4:30 – 6 PM ET

When Gut Feelings Aren’t Helping, Revisited: Navigating Complicated Ethics in Neuromuscular Care in 2021 (CME Accredited Session)

John Brandsema
MD (Chair)
Alyssa Burgart
MD
Tom Crawford
MD
Barkri Elsheikh
MD
Julie Parsons
MD

Rare Disease Initiatives

Barry Byrne
MD, PhD (Chair)
Alan Beggs
PhD
Philip John (PJ) Brooks
PhD
Jane Larkindale
Dphil
Laura Brod Hameed

Reproductive Health, Family Planning & Parenting (CME Accredited Session)

Elicia Estrella
MS, CGC, LGC (Chair)
Deodonne Bhattarai
Parent
Judy Jackson
MS, LCGC
Sharyn Lincoln
MS, LCGC
Alexandrea Lisi
BA Biology
6 – 9 PM ET

Dedicated Exhibit Hall & Poster Session

This time is dedicated to viewing Exhibit Booths & Posters exclusively Exhibit Booths & Posters to be staffed in real time (if possible)

6:30 – 9 AM ET

Dedicated Poster Session

This time is dedicated to viewing Posters exclusively Posters to staffed in real time (if possible)

9 AM – 6 PM ET

Exhibit & Poster Hall Open

9 – 10:30 AM ET

Impact of COVID-19 on Neuromuscular Disease Care (CME Accredited Session)

Nicholas Johnson
MD, MS-Cl (Chair)
Kathy Mathews
MD
Diana Mnatsakanova
MD

Measuring Progression of Disease & Selecting Endpoints

Erik Henricson
PhD, MPH (Chair)
Tina Duong
PT, PhD
Chad Heatwole
MD, MS-Cl
Stan Nelson
MD
10:30 AM – 12 PM ET

Clinical Trials in the Age of COVID-19

Jeffrey Statland
MD (Chair)
Nicholas Johnson
MD, MS-Cl
Petra Duda
MD, PhD
Michelle Mellion
MD
Lauren Morgenroth
MS

Navigating SMA Treatment Decisions (CME Accredited Session)

Perry Shieh
MD, PhD (Chair)
John Brandsema
MD
Diana Castro
MD
12 – 1:30 PM ET

Industry Forum Lunch Presented by Biogen

Current and Unmet Needs of Patients With SMA Across the Lifespan

Industry Forum Lunch Presented by PTC Therapeutics

Duchenne Muscular Dystrophy: Advancing the Body of Evidence for Corticosteroid Treatment

Industry Forum Lunch Presented by Sarepta Therapeutics

Development of AAVrh74 Micro-dystrophin Gene Transfer Therapy for Duchenne

1:30 – 3 PM ET

Telehealth in Neuromuscular Disease Care (CME Accredited Session)

Mario Saporta
MD, PhD, MBA, FAAN (Chair)
Gyula Ascadi
MD, PhD
Matthew Caraher
PR, DPT
Volkan Granit
MD
Michael Shy
MD

Therapies Targeting Pulmonary Function in Neuromuscular Disease (CME Accredited Session)

Jonathan Finder,
MD (Chair)
John Coleman
Ill, MD
Hank Mayer
MD
3 – 4:30 PM ET

Case Studies in Access to New Therapies (CME Accredited Session)

Claudia Chiriboga
MD, MPH (Chair)
Anne Connolly
MD, FAAN
Basil Darras
MD
Matt Harms
MD

A Multidisciplinary Approach to Clinical Trials; The Role of Physical and Occupational Therapists in Neuromuscular Research (CME Accredited Session)

Sally Dunaway Young
PT, DPT (Chair)
Jacqueline Montes
PT, Ed (Chair)
Kate Eichinger
PhD, DPT
Tim Estilow
OTR/L
Sara Feldman
PT, DPT, ATP
4:30 – 6 PM ET

Cardiomyopathy Across the Neuromuscular Disease Spectrum (CME Accredited Session)

Linda Cripe
MD (Chair)
Andreas Barth
MD, PhD, FAHA
Kan Hor
MD
Chet Villa
MD

Ethical Considerations in Gene Therapy Trials (CME Accredited Session)

Kevin Flanigan
MD (Chair)
Diana Bharucha-Goebel
MD
Barry Byrne
MD, PhD
6 – 9 PM ET

Dedicated Exhibit Hall Session

This time is dedicated to viewing Exhibit Booths exclusively Exhibit Booths to be staffed in real time (if possible)

6:30 AM – 9 AM ET

Dedicated Exhibit Hall & Poster Session

This time is dedicated to viewing Exhibit Booths & Posters exclusively Exhibit Booths & Posters to be staffed in real time (if possible)

9:00 AM – 6 PM ET

Exhibit Hall & Poster Session Open

9 AM – 12 PM ET

Oral Presentations from Abstract Submissions Part I

9:00 AM-9:15 AM

WVE-004, an Investigational Stereopure Antisense Oligonucleotide for the Treatment of Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia

Yuanjing Liu MD, PhD

9:15 AM-9:30 AM

Slc20a1/Pit1 and Slc20a2/Pit2 are Essential for Normal Skeletal Myofiber Function and Survival

Clemens Bergwitz, MD

9:30 AM- 9:45 AM

Resolution of Causality in Myotonic Dystrophy, Type 1 (DM1) via a PATrOL™-Enabled Therapy

Dietrich Stephan, Phd

9:45 AM-10:00 AM

AAV Gene Therapy for TNNT1-Associated Nemaline Myopathy

Eleonora D ’Ambrosio, md

10:00 AM-10:15 AM

Dendrimer-Conjugated GCPII Inhibitor Selectively Targets Activated Macrophages in SOD1G93A Muscle Which Delays Functional Loss and Denervation

Carolyn Tallon, Phd

10:15 AM- 10:30 AM

Galectin-1 Improves Sarcolemma Repair and Decreases Inflammatory Response in LGMD2B Models

Pam Van Ry

10:30 AM-10:45 AM

Micro-dystrophin Gene Therapy Delivery and Therapeutic Plasma Exchange in Non-Human Primates

Ellyn Peterson

10:45 AM -11:00 AM

A Hotspot in CAPN3 as the Likely Cause of Autosomal-Dominant Calpainopathy (LGMDD4)

Michelle Allen-Sharpley MD, PhD

11:00 AM-11:15 AM

Molecular Markers of Clinical Severity in Becker Muscular Dystrophy from a Remote Study

Utkarsh Dang, PhD

11:15 AM-11:30 AM

A Pilot Study Using a Novel Wearable Sensor to Examine Physical Activity in Individuals with Charcot Marie Tooth Disease 1A

Katy Eichinger PT, PhD

11:30 AM- 11:45 AM

Quantitative Muscle Analysis in FSHD Using Whole-Body MRI: Composite Muscle Measurements for Cross-Sectional Analysis

Michelle Mellion, MD
12 – 1:30 PM ET

Industry Forum Lunch Presented by NS Pharma

A Treatment Option for Patients with Duchenne Muscular Dystrophy Amenable to EXON 53 Skipping

Industry Forum Lunch Presented by Sanofi

The Challenges of Newborn Screening for Neuromuscular Disorders The Pompe Disease Experience

Industry Forum Lunch Presented by Solid Biosciences

Real World Outcome Measures in Duchenne Muscular Dystrophy: Current and Novel Assessments of Meaningful Patient Benefit

1:30 – 6 PM ET

Oral Presentations from Abstract Submissions Part II

1:30 PM-1:45 PM

Self and Parent Perceptions of Behavior and Quality of Life in Boys with Duchenne Muscular Dystrophy

Anna Jesus, MD Mbe

1:45 PM- 2:00 PM

Predictors of Functional Outcomes in Patients with Facioscapulohumeral Muscular Dystrophy

Natalie Katz, MD PhD

2:00PM-2:15 PM

Apitegromab, a Novel High-Affinity Anti-proMyostatin Monoclonal Antibody for Treating Spinal Muscular Atrophy: Results of a Phase 2 Interim Analysis

Amy Place, PhD, MBA, MS, RD, CLT

2:15 PM-2:30 PM

Predictive Factors of Nusinersen Treatment Response in Infantile-onset SMA: Results from the ENDEAR/SHINE Studies

Laurent Servais, MD, PhD

2:30 PM-2:45 PM

Kinetics of Early Antibody Response and Immunotoxicity Following Zolgensma for the Treatment of SMA Type 1

Stephanie Salbarria

2:45 PM-3:00 PM

SUNFISH Part 2: 24-Month Efficacy and Safety of Risdiplam in Patients with Type 2 or Non-Ambulant Type 3 Spinal Muscular Atrophy (SMA)

Maryam Oskoui, MD, MSc, FRCPC, FAAN

3:00 PM-3:15PM

Nusinersen in Children with SMA Who Received Onasemnogene Abeparvovec: Design of the Phase 4, Open-Label RESPOND Study

Julie Parsons, MD

3:15PM - 3:30PM

Long-Term Follow-Up (LTFU) of Onasemnogene Abeparvovec Gene Therapy in Spinal Muscular Atrophy (SMA)

Jerry R, Mendell, MD

3:30PM - 3:45PM

A Randomized, Double-Blind, Placebo-Controlled, Gene-Delivery Clinical Trial of rAAVrh74.MHCK7. Micro-Dystrophin for Duchenne Muscular Dystrophy

Jerry R. Mendell, MD

3:45PM-4:00PM

Lower Long-Term Risk of Death or Permanent Ventilation and First Hospitalization Among Participants with ALS Receiving AMX0035 in the CENTAUR Trial

Sabrina Paganoni MD, PhD

4:00PM-4:15PM

IGNITE-DMD: Phase I/II Ascending Dose Study of Single IV Infusion of SGT-001 Microdystrophin Gene Therapy for DMD-One Year Efficacy and Safety Results

Barry Byrne, MD, PhD

4:15PM-4:30PM

Onasemnogene Abeparvovec Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with 3 Copies of SMN2

Kevin Strauss, MD

4:30PM-4:45PM

Safety, β-sarcoglycan Expression, and Functional Outcomes from Systemic Gene Transfer of rAAVrh74.MHCK7.SGCB in Limb Girdle Muscular Dystrophy Type 2E

Louise Rodino-Klapac, phD

4:45PM-5:00PM

Safety and Efficacy of PF-06939926 Gene Therapy in boys with Duchenne Muscular Dystrophy: Update on data from the Phase 1b Study

Beth Belluscio MD, PhD

5:00PM-5:15PM

Early Intervention with Edaravone in Study 19 was Associated with Decreased Hospitalization, Tracheostomy, Ventilation, and Death in Patients with ALS

Benjamin Brooks, Md

5:15PM-5:30PM

2.5-years of Vamorolone Treatment in Duchenne Muscular Dystrophy: Results of an Open Label Long-Term Extension

Utkarsh Dang, PhD
7 PM ET

Meeting Adjourned

Sponsors

Strength of Life Partner

Circle of Strength

Impact

Friend of MDA

Frequently Asked Questions

Please click here to register for the conference.
Please click here to view the conference registration rates.
• This is an online event. You do not have to travel to any specific location. You can participate anywhere that you have Internet access. The event is accessible from desktop, mobile, and tablet devices.
We recommend using Chrome or Firefox.
Your login details will be emailed to you ahead of the event. You can use these details to access the online event.
No. You do not need to download or install any software to participate. You only need to have access to the internet.
Your conference login information is included in your registration confirmation email. You will also be re-notified of your login details prior to the conference.
Yes, you will be able to join a session late. Please note joining a CME session late may impact your CME credits.
Yes, all conference sessions will be made available on-demand within one week post the sessions live date.
All conference sessions will be made available on-demand within the virtual platform for one year.
Please click here to view the conference agenda.
vFairs runs on the cloud so you will not need to install software to access the virtual conference. All you will need is a standard web browser and a stable internet connection. We will also have a help desk available during the conference for any questions you may have.
Yes, please use the agenda on this webpage to add specific conference sessions to your calendar.
Yes, like any physical event, we will have a swag bag for you! You can add all the documents available to the online swag bag in your account and then email them to yourself.
The event will remain open 24/7 for one year. Therefore, you can attend whenever it is convenient for you.
The event will remain open 24/7 for one year. Therefore, you can attend whenever it is convenient for you.
      Monday, March 15, 2021 10 AM – 6 PM EST (Soft Opening)

      Tuesday, March 16, 2021, 6 PM - 9 PM EST

    Wednesday, March 17, 2021, 6 PM - 9 PM EST
The booth selection process will be on a first come, first-served basis. The Exhibit Hall will be located within the virtual event platform. Exhibitors can select from four different booth design options. Exhibit material deadlines and additional details will be provided to all exhibitors. For more information, please contact Nicole Camacho at ncamacho@mdausa.org.
Yes, all virtual exhibit booths will have a live chat feature where you can chat with the organization’s booth administrators.
Yes, MDA will provide exhibitors with detailed reports of all attendees that visited the virtual exhibit booth.
The virtual exhibit booths will be listed in alphabetical order by organization name.