Annual SMA Conference to Host Panels on NDA Process and SMA Drugs in Development

One of the most significant aspects of the Annual SMA Conference is the bringing together of families and researchers in the same event. The 2016 Annual SMA Conference is poised to set a record for the largest conference ever, with well over 1100 families and researchers already registered.

As the world’s largest gathering of SMA families and researchers, the conference is an ideal forum to come together and evaluate current developments in SMA research, and chart out the next steps toward a treatment and cure. The Saturday afternoon Research Q&A Session will be a pivotal opportunity to advance those goals.

Currently, there are 18 drugs in development, with six in clinical trials. With several of those drugs progressing into Phase 2 and Phase 3 clinical trials, we believe we are closer than ever to an approved treatment for SMA.

The final stage in gaining an approved treatment is a new drug application (NDA) with the FDA. With this in mind, we’ve gathered an expert panel who will walk our community through the NDA process, including a time for questions and answers.

Following that panel, representatives from all six programs currently in clinical trials will present updates, followed by another Q&A session.


Moderated Panel Discussion on the NDA Process

  • Annie Kennedy, Senior Vice President, Legislation & Public Policy, Parent Project Muscular Dystrophy
  • Douglas Kerr, MD, PhD, MBA, Global Neurology Development Lead, Shire
  • John T. Kissel, MD, Chair, Department of Neurology, The Ohio State University (Moderator)
  • Katherine Klinger, PhD, Sr. Vice President, Genetics and Genomics and Presidential Fellow, Genzyme Corporation
  • Thomas H. Murray, PhD, President Emeritus, The Hastings Center

Panel on SMA Drugs in Clinical Development

  • Stacy Rudnicki, MD, Director of Clinical Research and Development, Cytokinetics
  • Douglas M. Sproule, MD, MSc, Vice President, Clinical Development, AveXis Inc.
  • Wildon Farwell, MD, MPH, Senior Medical Director, Clinical Development Rare Diseases, Biogen
  • Omar Khwaja, MD, PhD, Head of Rare Disease, Research and Early Development, F. Hoffmann-La Roche
  • Eugene Schneider, MD, Executive Director, Clinical Development, Ionis Pharmaceuticals
  • Lawrence Charnas, MD, PhD, Sr. Translational Medicine Expert, Musculoskeletal Diseases, Novartis Institutes for Biomedical Research
  • Thomas Blaettler, MD, Global Clinical Development Team Leader, F. Hoffmann-La Roche

Our thanks to Biogen for their generosity as the presenting sponsor of the 2016 Annual SMA Conference.

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