Annual SMA Conference to Feature Panel of Leading SMA Drug Experts

The Annual SMA Conference will bring together well over a thousand families and researchers from around the world. During the conference, several sessions are set aside specifically for families and leading researchers to interact. One of the most important of these sessions is the Research Q&A Session that closes the conference.

A dozen experts will be present to update families on the latest drug programs and clinical trials. A wide range of academic research institutions, six pharmaceutical companies, and the FDA will be represented by the panelists. Currently, there are 17 drugs in development for SMA, and seven of those are in clinical trials—both the highest numbers ever. And the pace of progress is increasing, particularly in clinical trials.

As the world’s largest gathering of SMA families and researchers, the Annual SMA Conference is an ideal forum to come together and evaluate these and other current developments in SMA research, and chart out the next steps toward a treatment and cure.

In addition, Cure SMA will also be making the panel presentation available for online viewing after the conference, further expanding this important discussion.


Moderated Panel Discussion on SMA Clinical Trials

  • John Kissel, MD, Chair, Department of Neurology, The Ohio State University (moderator)
  • Richard Finkel, MD, Division Chief, Division of Neurology, Nemours Children’s Hospital
  • Katherine Klinger, PhD, Sr. Vice President, Genetics and Genomics and Presidential Fellow, Genzyme Corporation
  • Thomas H. Murray, PhD, Senior Research Scholar, President Emeritus, The Hastings Center
  • John Whyte, MD, MPH., Director of Professional Affairs and Stakeholder Engagement, Center for Drug Evaluation and Research, FDA

Panel on SMA Drugs in Clinical Development

  • Jinsy Andrews, MD, Director of Clinical Research and Development, Cytokinetics
  • Brian Kaspar, PhD, Grant Morrow III, MD, Endowed Chair in Pediatric Research, The Research Institute at Nationwide Children’s Hospital, Columbus Ohio
  • Wildon Farwell MD, MPH, Medical Director, Neurology Early Clinical Development, Biogen
  • Irene Gerlach, PhD, Project Team Leader Rare Diseases, F. Hoffmann-La
  • Eugene Schneider, MD, Executive Director, Clinical Development, Isis Pharmaceuticals
  • Emilie Voltz, PhD, Clinical Trials Leader in Neurosciences, Translational Medicine, Novartis Institutes for Biomedical Research

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