Register for the 2022 Clinical and Scientifc Conference

 

ALL CONFERENCE SESSION TIMES LISTED ARE IN CENTRAL STANDARD TIME

9:00 AM – 5:00 PM

Insights in Research Investor Summit (IRIS)

Add to Calendar3/13/2022 06:00 PM4/13/2022 10:00 PMAmerica/Mexico_CityInsights in Research Investor Summit (IRIS)Click here to join the virtual summit: https://2022mdavirtualconference.org/Online
6:00 PM – 8:00 PM

Welcome Reception in Exhibit Hall

Add to Calendar3/13/2022 06:00 PM4/13/2022 10:00 PMAmerica/Mexico_CityWelcome Reception in Exhibit HallClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
7:00 AM – 8:00 AM

Industry Forum Breakfast Sponsored by Edgewise

A Novel Approach to Protecting Dystrophic Muscle by Targeting Fast Muscle Myosin: EDG-5506

Add to Calendar3/14/2022 07:00 AM3/14/2022 8:00 AMAmerica/Mexico_CityIndustry Forum Breakfast Sponsored by EdgewiseClick here to join the virtual summit: https://2022mdavirtualconference.org/Online

Industry Forum Breakfast Sponsored by NS Pharma

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

Add to Calendar3/14/2022 07:00 AM3/14/2022 8:00 AMAmerica/Mexico_CityIndustry Forum Breakfast Sponsored by NS PharmaClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
8:30 AM - 9:30 AM

Opening and Keynote Address

Add to Calendar3/14/2022 08:30 AM3/14/2022 9:30 AMAmerica/Mexico_CityOpening and Keynote AddressClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
10:00 AM – 8:00 PM

Exhibit Hall and Poster Sessions Open

Add to Calendar3/14/2022 10:00 AM3/14/2022 8:00 PMAmerica/Mexico_CityExhibit Hall and Poster Sessions OpenClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
9:30 – 10 AM

Networking Break sponsored by Andelyn Biosciences

Add to Calendar3/14/2022 10:00 AM3/14/2022 8:00 PMAmerica/Mexico_CityNetworking Break sponsored by Andelyn BiosciencesClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
10:00 AM – 12:00 PM

Latest Developments Across the NMD Registry Data Landscape

Speaker ImageRayne Rodgers, MPH (Co-Chair)

Speaker ImageElisabeth Kilroy, PhD (Co-Chair)

Session will discuss real-world data in neuromuscular disease and its application to therapy development.

Add to Calendar3/14/2022 10:00 AM3/14/2022 12:00 PMAmerica/Mexico_CityLatest Developments Across the NMD Registry Data LandscapeClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
12:00 PM – 01:30 PM

Industry Forum Lunch Presented by Biogen

Supporting Patients with SPINRAZA ® (nusinersen): Learn More About Our Clinical Trial Program and the Latest Real-World Evidence


Industry Forum Lunch Presented by Pfizer

Innovation for Rare Neuromuscular Diseases


Industry Forum Lunch Presented by Sarepta

What Lies Beneath: Uncovering the Clue for Earlier Diagnosis and Care of Young Individuals with Duchenne

Add to Calendar3/14/2022 12:00 PM3/14/2022 01:30 PMAmerica/Mexico_CityIndustry Forum Lunch Presented by BiogenClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
1:30 PM – 2:30 PM

Lab to Life: LGMD

Speaker ImageNicolas Johnson, MD, MS-CI (Chair)


Session will review advances in translational research in LGMD. Speakers will discuss steps to improve genetic diagnosis, development of small molecules, and advances in gene replacement therapy.

Add to Calendar3/14/2022 01:30 PM4/14/2022 2:30 PMAmerica/Mexico_CityNicolas Johnson, MD, MS-CI (Chair)Click here to join the virtual summit: https://2022mdavirtualconference.org/Online
1:30 PM – 3:30 PM

New Gene Discoveries in NMD

(CME Accredited Session)
Speaker ImageAlan Beggs, PhD (Chair)

Speaker ImageAngela Lek, PhD (Co-Chair)

Session will provide an overview of the rich and rapidly evolving body of knowledge in this area, with talks focused on collaborative and crowd-sourcing approaches to information sharing for new disease gene discovery, the latest on genetic heterogeneity among inherited peripheral neuropathies, approaches to undiagnosed neuromuscular diseases, and new genetic modifiers, which can impact the course of a patient’s disease.

Add to Calendar3/14/2022 01:30 PM3/14/2022 3:30 PMAmerica/Mexico_CityNew Gene Discoveries in NMDClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
2:30 PM – 3:30 PM

Lab to Life: FSHD

Speaker ImageJeffrey Statland, MD (Chair)


Session explores implementation of targeted therapies in clinical trials, our molecular understanding of FSHD, and how we measure changes in future clinical trials

Add to Calendar3/14/2022 02:30 PM3/14/2022 3:30 PMAmerica/Mexico_CityLab to Life: FSHDClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
03:30 pm – 04:00 PM

Networking Break sponsored by Hill-Rom

4:00 PM - 5:00 PM

Lab to Life: MG

Speaker ImageHenry Kaminski, MD (Chair)


Session will review the journey of treatment to patient from identification of unmet need to prescription of the drug and access for all patients.

Add to Calendar3/14/2022 04:00 PM3/14/2022 5:00 PMAmerica/Mexico_CityLab to Life: MGClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
4:00 PM - 5:00 PM

Functional/PROs/Composite Clinical Trial Endpoints in DMD

(CME Accredited Session)
Speaker ImageDr. Erik Henricson, PhD, MPH (Chair)


Session will explore the uses and intersections between Clinically Reported Outcome tools, Person-Reported Outcome tools, and single- and multi-domain composite endpoints in DMD clinical research. Emphasis will be placed on how tools create a continuum of measures that can be extended from observed clinical task performance, to individual perceptions of ability, to community mobility and participation and life satisfaction

Add to Calendar3/14/2022 04:00 PM3/14/2022 5:00 PMAmerica/Mexico_CityFunctional/PROs/Composite Clinical Trial Endpoints in DMDClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
5:00 PM – 6:00 PM

Lab to Life: Congenital Myopathies/Dystrophies

Speaker ImageChamindra Konersman, MD (Chair)


Session will provide understanding of the disease spectrum, genetics and phenotypic complexity of congenital myopathies and congenital muscular dystrophies. Understand current and future strategies for disease modifying treatments and preparing for meaningful clinical trial outcome measures.

Add to Calendar3/14/2022 05:00 PM3/14/2022 6:00 PMAmerica/Mexico_CityLab to Life: Congenital Myopathies/DystrophiesClick here to join the virtual summit: https://2022mdavirtualconference.org/Online



Best Practices in Respiratory Care

(CME Accredited Session)

Speaker ImageBrad Troxler, MD (Chair)


Session will provide a forum to learn about the latest advancements in respiratory care for patients with NMD. Use of advanced airway clearance techniques and positive airway pressure devices will be discussed. The impact of respiratory care on patient with NMD will be shared.

Add to Calendar3/14/2022 05:00 PM3/14/2022 6:00 PMAmerica/Mexico_CityLab to Life: Congenital Myopathies/DystrophiesClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
6:00 PM – 8:00 PM

Poster & Networking Reception

Add to Calendar3/14/2022 06:00 PM3/14/2022 8:00 PMAmerica/Mexico_CityPoster & Networking ReceptionClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
7:00 AM – 8:00 AM

Industry Forum Breakfast Sponsored by Amylyx

Amyotrophic Lateral Sclerosis (ALS) in Depth: Key Topics in Pathophysiology and Diagnosis


Industry Forum Breakfast Sponsored by Fulcrum

Measuring Impact on Disease Progression in FSHD

Add to Calendar3/15/2022 07:00 PM3/15/2022 8:00 PMAmerica/Mexico_CityIndustry Forum Breakfast Sponsored by AmylyxClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
8:30 AM - 10:00 AM

Mitochondrial Interventions: Small Molecule and Gene Therapy

Speaker Image Carlos Moraes, PhD (Chair)


Session will explore a few approaches with great clinical potential that use small molecules or genetic manipulation.

Add to Calendar3/15/2022 08:30 AM3/15/2022 10:00 AMAmerica/Mexico_CityMitochondrial Interventions: Small Molecule and Gene TherapyClick here to join the virtual summit: https://2022mdavirtualconference.org/Online



Promoting Diversity in Research

Speaker ImageSusan Apkon, MD (Chair)


Session will provide a greater understanding of the barriers to participation in clinical research for patients from diverse backgrounds and opportunities to improve their involvement. Panelists will share their diverse experience and perspectives as clinicians, family members, and industry partners.

Add to Calendar3/15/2022 08:30 AM3/15/2022 10:00 AMAmerica/Mexico_CityPromoting Diversity in ResearchClick here to join the virtual summit: https://2022mdavirtualconference.org/Online



Telehealth Learnings in Clinical Care and Research: Learnings from the Pandemic

(CME Accredited Session)
Speaker ImageDustin Nowacek, MD (Chair)


Session will discuss telehealth experiences, identify the benefits of telehealth, and recognize the limitations of virtual care

Add to Calendar3/15/2022 08:30 AM3/15/2022 10:00 AMAmerica/Mexico_CityTelehealth Learnings in Clinical Care and Research: Learnings from the PandemicClick here to join the virtual summit: https://2022mdavirtualconference.org/Online



When Gut Feelings Aren’t Helping, Revisited: Navigating Complicated Ethics in Neuromuscular Care in 2022

(CME Accredited Session)
Speaker ImageJohn Brandsema, MD (Chair)


Session aims to review cases from neuromuscular practice that have been complicated ethically and generate an open forum for discussion of how to best navigate similar situations when they arise

Add to Calendar3/15/2022 08:30 AM3/15/2022 10:00 AMAmerica/Mexico_CityWhen Gut Feelings Aren’t Helping, Revisited: Navigating Complicated Ethics in Neuromuscular Care in 2022Click here to join the virtual summit: https://2022mdavirtualconference.org/Online
10:00 AM – 8:00 PM

Exhibit Hall and Poster Sessions Open

Add to Calendar3/15/2022 10:30 AM3/15/2022 8:00 PMAmerica/Mexico_CityExhibit Hall and Poster Sessions OpenClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
10 – 10:30 AM

Networking Break sponsored by Brainstorm Therapeutics

Add to Calendar3/15/2022 10:30 AM3/15/2022 8:00 PMAmerica/Mexico_CityNetworking Break sponsored by Brainstorm TherapeuticsClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
10:30 AM – 12:00 PM

Small Molecule Approaches to Overcoming Muscle Wasting

Speaker ImageHelen Blau, PhD (Co-Chair)

Speaker ImageMichael Rudnicki, OC, PhD, FRS, FRSC (Co-Chair)


Session will discuss small molecule drugs that counter muscle loss and show promise in treating SMA, DMD, and BMD.

Add to Calendar3/15/2022 10:30 AM3/15/2022 12:00 PMAmerica/Mexico_CitySmall Molecule Approaches to Overcoming Muscle WastingClick here to join the virtual summit: https://2022mdavirtualconference.org/Online



More Data, Lower Burden – Digital Outcome Measures in Neuromuscular Disease Therapy Development

Speaker ImageJames Berry, MD, MPH (Chair)


Session will explore digital outcomes measures in neuromuscular disease therapy development

Add to Calendar3/15/2022 10:30 AM3/15/2022 12:00 PMAmerica/Mexico_CityMore Data, Lower Burden – Digital Outcome Measures in Neuromuscular Disease Therapy DevelopmentClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
10:30 AM – 12:00 PM

Natural History and Trial Readiness for Children with Charcot Marie Tooth Disease

(CME Accredited Session)

Speaker Image Michael Shy, MD (Chair)


Session will provide an update on natural history studies, a description of pediatric patient-reported outcomes assessment evaluating the self-reported quality of life for children with CMT, approaches to evaluating and managing children with CMT, and approaches in clinical trial design in children with CMT and other rare forms of NMD


Facilitating Multi-Practice Collaboration to Optimize Patient Care

(CME Accredited Session)

Speaker Image Lora Clawson, MSN, CRNP (Chair)


Session will provide an overview of MDA clinical programs & collaborators; describe how these collaborators are engaged; and describe the challenges and successes in maintaining these important collaborations.

Add to Calendar3/15/2022 10:30 PM3/15/2022 12:00 PMAmerica/Mexico_CityNatural History and Trial Readiness for Children with Charcot Marie Tooth DiseaseClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
12:00 PM – 01:30 PM

Industry Forum Lunch Presented by Genentech

Explore the Science for an Approved Spinal Muscular Atrophy Treatment and Hear from People Living with SMA


Industry Forum Lunch Presented by PTC

Throughout the Disease Continuum: Corticosteroid Treatment in Duchenne Muscular Dystrophy


Industry Forum Lunch Presented by Sarepta

The Intersection of Science and Medicine – An Investigational rAAVrh74-based Gene Transfer Therapy for Individuals with Duchenne

Add to Calendar3/15/2022 12:00 PM3/15/2022 01:30 PMAmerica/Mexico_CityIndustry Forum Lunch Presented by GenentechClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
1:30 PM – 2:30 PM

Building MD Team in NMD

(CME Accredited Session)
Speaker ImageYaacov Anziska, MD (Chair)


Session will address experiences from a range of specialists about coordinating with the neurologist to provide the best care for our MDA neuromuscular patients

Add to Calendar3/15/2022 1:30 PM3/15/2022 02:30 PMAmerica/Mexico_CityBuilding MD Team in NMDClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
1:30 PM – 3:30 PM

Personalized Medicine/Regulatory Approaches for Rare Disease

(CME Accredited Session)
Speaker ImageTeepu Siddique, MD (Chair)


Progress in Brain Interface Technology

Speaker ImageDaniel Rubin, MD, PhD (Co-Chair)

Speaker ImageLeigh Hochberg, MD, PhD (Co-Chair)


Session will review recent progress, with leaders in the field sharing their vision for the clinical applications and future of these important technologies


Treatment Decisions for Disorders with Multiple Therapies

(CME Accredited Session)

Speaker ImageJulie Parsons, MD (Chair)


Session will discuss FDA approved therapies for SMA, Duchenne Muscular Dystrophy, ALS, and Myasthenia Gravis. Strategies for medical decision making and treatment selection will be reviewed

Add to Calendar3/15/2022 1:30 PM3/15/2022 02:30 PMAmerica/Mexico_CityPersonalized Medicine/Regulatory Approaches for Rare DiseaseClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
2:30 – 3:30 PM

The Resilient Practitioner

Add to Calendar3/15/2022 04:00 PM3/15/2022 06:00 PMAmerica/Mexico_CityThe Resilient PractitionerClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
3:30 PM - 4:00 PM

Networking Break

4:00 PM - 6:00 PM

CRISPR – The Next Generation

Speaker ImageEric Olson, PhD (Chair)


Session will focus on recent advances and future challenges in translating CRISPR gene editing for genetic myopathies from animal models toward human translation.

Clinical Trial Design for Rare Neuromuscular Disorders – Where Are We Headed?

Speaker ImageDiana Bharucha-Goebel MD, MD (Chair)


Session will explore issues common to trials in rare disease such as phenotypic pleiotropy, genetic heterogeneity, combination therapies and n=1 trials.

Impact of Gene Transfer Therapy on Long-Term Multidisciplinary Care

(CME Accredited Session)

Add to Calendar3/15/2022 04:00 PM3/15/2022 06:00 PMAmerica/Mexico_CityImpact of Gene Transfer Therapy on Long-Term Multidisciplinary CareClick here to join the virtual summit: https://2022mdavirtualconference.org/Online

Business Operations for NMD Clinics

(CME Accredited Session)


Four clinicians will provide their perspective including costs, reimbursement, obtaining assistance and grant funding, hurdles, research, the role of telemedicine, altruism, and future recommendations.

Add to Calendar3/15/2022 04:00 PM3/15/2022 06:00 PMAmerica/Mexico_CityBusiness Operations for NMD ClinicsClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
6:00 PM - 8:00 PM

Exhibits & Poster Networking Reception (Exhibit Hall)

Add to Calendar3/15/2022 6:00 PM3/15/2022 08:00 PMAmerica/Mexico_CityExhibits & Poster Networking Reception (Exhibit Hall)Click here to join the virtual summit: https://2022mdavirtualconference.org/Online

New, Novel and Noteworthy: NMD Highlights

We are thrilled to have had an overwhelming number of incredible abstract submissions as we reconvene in 2022. To that end, we have redesigned our Clinical Trail Presentations to include a wider range of topics which also allows for event more live presentations selected directly from the abstract submissions. This is a final day not to be missed!

8 AM – 12:25 PM

Clinical Trial Out Comes

Add to Calendar3/16/2022 08:00 AM3/16/2022 12:25 PMAmerica/Mexico_CityClinical Trial Out ComesClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
8:30 AM – 11:30 AM

Novel Therapeutic Approaches to Treat Neuromuscular Diseases

Add to Calendar3/16/2022 08:30 PM3/16/2022 11:30 PMAmerica/Mexico_CityNovel Therapeutic Approaches to Treat Neuromuscular DiseasesClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
11:15 AM – 12:00 PM

Insights into Neuromuscular Disease Mechanism

Add to Calendar3/16/2022 11:15 AM3/16/2022 12:00 PMAmerica/Mexico_CityInsights into Neuromuscular Disease MechanismClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
8:30 AM – 12:20 PM

Strategies to Improve Oligonucleotide Delivery

Add to Calendar3/16/2022 08:30 AM3/16/2022 12:20 PMAmerica/Mexico_CityStrategies to Improve Oligonucleotide DeliveryClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
10:00 AM – 11:20 AM

Towards Clinical Trials: IND-enabling Studies and Outcome Measures

Add to Calendar3/16/2022 10:00 AM3/16/2022 11:20 AMAmerica/Mexico_CityTowards Clinical Trials: IND-enabling Studies and Outcome MeasuresClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
11:25 AM – 12:20 PM

Diagnosis and Clinical Management for NMDs

Add to Calendar3/16/2022 11:25 AM3/16/2022 12:20 PMAmerica/Mexico_CityDiagnosis and Clinical Management for NMDsClick here to join the virtual summit: https://2022mdavirtualconference.org/Online
1 PM – 03:50 PM

Highlight Sessions

Add to Calendar3/16/2022 01:00 PM3/16/2022 03:50 PMAmerica/Mexico_CityHighlight SessionsClick here to join the virtual summit: https://2022mdavirtualconference.org/Online

Sponsors

Strength of Life Partner

Circle of Strength

Impact Sponsors

Empowerment Sponsors

 

Champion Sponsors

       

Friends of MDA