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Improving Outcomes in Spinal Muscular Atrophy: Best Practices for Clinicians and Caregivers

Now Available On Demand

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Produced in collaboration with CureSMA and NORD
Support for this patient education program was provided by Biogen.

Spinal Muscular Atrophy (SMA) is a genetic disease that takes away a person’s ability to walk, eat, and breathe. SMA primarily affects children, and the most common form is often fatal early in life – in fact, of all genetic diseases, SMA is the leading cause of death for children under two. Fortunately, huge progress has been made in the last 10 years: there is now an approved medicine that targets the underlying cause of SMA, and we’re very close to reaching more breakthroughs that will save and extend lives.

Join this special educational program to hear from other families facing SMA and learn along with some of the nation’s leading experts. The panel discusses:

  • Key features of SMA, and the genetic basis of the condition
  • What tests are done throughout the course of disease, and what their results mean
  • Treatment options for SMA, and how disease-related complications are addressed

Save a virtual seat to attend live or watch on-demand.

 

Speakers

Basil T. Darras, MD

Boston Children's Hospital

Basil T. Darras, MD, is the Joseph J. Volpe Professor of Neurology at Harvard Medical School, and associate neurologist-in-chief, chief of the Division of Clinical Neurology, and director of the neuromuscular program at Boston Children’s Hospital. His primary research focus is on pediatric neuromuscular disorders, specifically molecular genetics, diagnostics, and therapeutics of many myopathies, including spinal muscular atrophy (SMA), which has resulted in over 190 original reports in peer-reviewed journals and over 80 chapters, reviews, and editorials. Additionally, he receives frequent invitations to lecture both nationally and internationally. Dr Darras is editor in chief for the second edition of Neuromuscular Disorders of Infancy, Childhood, and Adolescence: A Clinician’s Approach.

Vanessa Battista, MS, RN, CPNP

Children's Hospital of Philadelphia

Vanessa Battista, MS, RN, CPNP-PC, is a board-certified pediatric nurse practitioner and a member of the neuromuscular team at The Children’s Hospital of Philadelphia. She teaches at the local, national, and international level, and is a member of the ELNEC pediatric faculty, for which she was the recipient of the Award for Excellence. Ms Battista is an active reviewer for publications and has authored several book chapters and articles on various aspects of pediatric palliative care and neuromuscular disease. She serves as a board member for organizations that provide services for patients and families living with life-threatening diseases. Ms Battista is currently obtaining her Master of Business Administration and Executive Doctor of Nursing Practice from Johns Hopkins University.

Danyelle Sun, MSW

Cure SMA

Dany Sun, the social manager at Cure SMA, lives in Greendale, Wisconsin with her family. Dany worked in mental and behavioral health programs after receiving her master’s degree in social work from the University of Wisconsin-Milwaukee, and eventually managed a variety of programs that serve youth and adults. After both of her children were diagnosed with Spinal Muscular Atrophy in 2013, Dany became involved in advocacy efforts of those in the SMA community, as well as the overall rare disease community, by supporting social-emotional and daily living needs at local and national levels. Dany's passion is advocacy and empowerment for individuals and families experiencing challenges in life, especially those within the disability community.

Rachel

Caregiver Presenter

Bio

Patrick

Caregiver Presenter

Bio

 

Partners and Supporters

  • CureSMA
  • NORD