MDA’s 2018 Clinical Conference Will Focus on Clinical Management in the Rapidly Changing Neuromuscular Disease Landscape

Next week, MDA will hold its 2018 Clinical Conference at the Hyatt Regency Crystal City in Arlington, Va., March 11-14. The meeting is the preeminent gathering of MDA Care Center multidisciplinary health care providers from across the country, as well as medical and scientific experts from academic, private practice and government institutions, and representatives from the biotech and pharma industry.

Part of MDA’s prestigious annual conference series, the 2018 Clinical Conference reflects MDA’s commitment and efforts to enhance the communication of new medical advances and information relating to the delivery of best-in-class care for individuals with neuromuscular disease.

Unprecedented times

The conference takes place at an unprecedented time in the history of MDA and the neuromuscular disease field, with the approval by the U.S. Food and Drug Administration of six drugs — four of them that can trace their origins to MDA-funded research — in the last three years for the treatment of neuromuscular diseases in MDA’s program.

Advances such as these — both in supportive and, in some cases, disease-modifying treatments — along with refinement of the multidisciplinary care model and standardization of best practices, are helping individuals with neuromuscular diseases live longer and enjoy a better quality of life.

MDA’s 2018 conference will act as a catalyst to spur more medical breakthroughs as it serves to help enable health care providers to understand the latest strategies and findings in core approaches to medical management, increase knowledge about what’s happening with clinical trials, gain insight into medical best practices to improve outcomes and increase patient satisfaction, and gain enhanced understanding of diagnostic tools. This important event also serves as a collaborative platform for clinicians, researchers and industry professionals to connect and share insights and perspectives on how we can work together to maximize collective impact.

Unprecedented engagement

This year’s conference is the largest and most comprehensive conference of this nature in MDA history. Nearly 700 attendees from MDA Care Centers and Care Affiliates, academic laboratories, clinics and the biotech and pharma industries are expected to attend, with more than 50 platform presentations and panel discussions and more than 85 poster presentations.

Keynote and highlights

Keynote Speaker Jerry Mendell, M.D., will open the conference on Sunday, March 11.

Mendell is the founder of the Clinical Translational Gene Therapy Program at the Research Institute at Nationwide Children’s Hospital, and he holds professorships in neurology, pediatrics, pathology and physiology and cell biology at The Ohio State University.

He is a clinician scientist, directing laboratory projects and carrying these to the bedside while at the same time caring for people with neuromuscular diseases in the clinic. He has published more than 350 articles with a focus on neuromuscular disease and authored books on muscle and nerve disease.

Mendell was the first to perform gene therapy for Duchenne muscular dystrophy (DMD) in March 2007, and started a gene therapy study in limb-girdle muscular dystrophy type 2D in November 2007, demonstrating success for the first time. Mendell is also the lead investigator on the first gene therapy trial for spinal muscular atrophy (SMA), which launched in 2014. He will open the conference with a presentation titled “Changing the Playing Field for Children with Neuromuscular Disease: Clinical Gene Therapy for SMA and DMD.”

Other notable presentations showcase the use of technology in neuromuscular disease care. These include:

  • Murray Aitken, MBA, from IQVIA Institute for Human Data Science, who will speak about the role of digital health in neuromuscular disease
  • Jane Yu, M.D., Ph.D., from Watson Health Life Sciences Solutions, who will talk about using cognitive computing to accelerate discovery in neurological disorders
  • Jeffrey Rothstein, M.D., Ph.D., from Johns Hopkins University School of Medicine, who will talk about Answer ALS, a population-based program to identify ALS (amyotrophic lateral sclerosis) subgroups
  • James D. Berry, M.D., from Massachusetts General Hospital, who will discuss telemedicine in ALS clinical care

Conference content

Presentations for neuromuscular health care professionals will focus on many topics, including the following:

  • What’s new in — ALS, DMD, facioscapulohumeral muscular dystrophy (FSHD), SMA, myotonic dystrophy (DM) and myasthenia gravis (MG)
  • Advances in personalized medicine — genome editing in DMD, scaling up genomic diagnosis and developments in digital health
  • Best practices in — cardiac care, physical therapy in SMA, bone health, health care technology, dysphagia and nutrition, and care coordination
  • Newborn screening — the latest insights on newborn screening in DMD, Pompe disease and SMA, and public policies that impact this important program
  • Augmentative communication — early engagement in assessment, system design and implementation in ALS
  • Transitioning from pediatric to adult-based health care — exploring how best to help patients shift from the pediatric to adult clincal care environment
  • Respiratory health — assisted ventilation at home, transitioning to home mechanical ventilation and respiratory management with airway clearance devices
  • Clinical trials — reports on data from clinical trials in ALS, DM, DMD, LAMA2 and COL6-related dystrophy, Pompe disease and SMA 

Conference sponsors

MDA is grateful to the following companies for their support of the 2018 Clinical Conference:

  • Strength for Life supporters — Biogen, PTC Therapeutics
  • Muscle Champion supporters — AveXis, Santhera Pharmaceuticals, Inc., Sarepta Therapeutics

Collaboration and communication

The care management landscape for neuromuscular diseases continues to evolve rapidly, making it more important than ever for physicians, allied care professionals and other key stakeholder groups, including industry, academia and government, to stay up-to-date on the current standards of care.

We know that communication is key to ensuring optimal care and quality of life for individuals with these disorders, and that is why MDA is dedicated to unifying the neuromuscular disease community and convening conferences such as this one to facilitate collaboration and dialogue among the world’s top health care professionals. The conference and the profound impact it will have on care in the neuromuscular disease space exemplify MDA’s commitment to strength, independence and life.

To read recaps of each day of the conference, click here.