Events Calendar

Gene Replacement Therapy in Spinal Muscular Atrophy

Tuesday, July 10, 2018

12:30–1:30 PM
Hilton Vienna Hotel
Congress Park 2
Vienna, Austria


Program Overview:
As new therapies emerge for spinal muscular atrophy that include gene replacement and gene modifying therapies, clinicians need to consider the expectations for these treatments. Experts, Dr. Eugenio Mercuri, Dr. Richard Finkel and Dr. Gyula Acsadi, will hold a roundtable discussion about the latest information in these emerging therapies and their implications on the clinical disease state. Discussion topics will include the durability of new and emerging treatments and changes to the standards of care for patients with SMA. Lessons from these treatments may have potential applications in the treatment of other neuromuscular diseases.

Eugenio Mercuri, MD, PhD (Chair)
Professor of Pediatric Neurology
Catholic University of the Sacred Heart
Rome, Italy

Richard S. Finkel, MD
Division Chief of Neurology
Pediatric Neurology Nemours Children’s Hospital
Orlando, Florida, United States

Gyula Acsadi, MD, PhD
Division Head of Neurology and Rehabilitation
Professor of Pediatric Neurology
Connecticut Children’s Medical Center
University of Connecticut Medical School
Hartford, Connecticut, United States

Topics to Include:

  • Genetics and Diagnostic Criteria of SMA
  • Emerging Data in SMA: Gene Replacement and Gene Modifying Therapies
  • Durability of New and Emerging Treatments
  • Implications for Standards of Care
  • Potential Applications to Other Neuromuscular Diseases

Target Audience:
This activity is intended for neuromuscular specialists and neurologists.

Accreditation Statement
The France Foundation is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Credit Designation
The France Foundation designates this live activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Commercial Support Acknowledgment
This activity is supported by an educational grant from AveXis, Inc.

This presentation was approved by the Scientific Program Committee as an independent activity held in conjunction with the 15th International Congress on Neuromuscular Diseases. This presentation is not sponsored or endorsed by ICNMD 2018.

Hilton Vienna Hotel

1 Am Stadtpark 1030
Vienna, Austria,

Location Website


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