The Family Friendly Research Poster Session allows for one-on-one interactions between families and researchers. Cure SMA has invited researchers, who are attending SMA Researcher Meeting, to present family friendly research posters. During the event, families are encouraged to rotate to the different posters to ask questions and learn directly from the researchers involved in each of the projects being presented.


  1. Kristina Gelblin from F. Hoffmann-La Roche on “Partnering with Patients in the Roche and Genentech SMA development program”. 
  2. Ksenija Gorni MD PhD, Tim Seabrook PhD, and Sangeeta Jethwa MD from F. Hoffmann-La Roche on “Roche and Genentech SMA program advancing in clinical development”. 
  3. Dylan Trundell PhD from F. Hoffmann-La Roche on “Living with SMA-Individuals with SMA and their caregivers share their experiences”.
  4. Kimberly Long PhD from Scholar Rock on “Specific inhibition of myostatin activation improves muscle and bone phenotypes in models of SMA therapy”.
  5. Fernanda Boulos MD and Herve Jullien de Pommerol PhD from Novartis on “Introduction to Novartis”. 
  6. Douglas M. Sproule MD from AveXis, Inc. on “Understanding Gene Replacement Therapy and the potential to help people with monogenic diseases”.
  7. Zaida Alipio-Gloria from Calibr on “Innovative screening approaches to ID the next generation of SMN-inducing compounds”. 
  8. Lisa Belter MPH from Cure SMA on “Cure SMA Membership: Findings from the 2018 Membership Survey”.
  9. Rosangel Cruz MA from Cure SMA on “Benefit-Risk Survey Results for Spinal Muscular Atrophy-Informing Drug Development in SMA”.
  10. Mary Schroth MD and Page Kirkpatrick MHA, MBA from Cure SMA on “Cure SMA Care Center Network and SMA Clinical Data Registry: Using data to improve care”.
  11. Jackie Glascock PhD from Cure SMA on “Development of a Treatment Algorithm for Infants with SMA Identified Via Newborn Screening”.
  12. Bakri Elsheikh MD from Ohio State University on “Nusinersen in adults with SMA: The Ohio State Experience”.
  13. Kathryn J. Swoboda MD, Jin Yun Helen Chen LGC, and Ren Zhang MPH from Massachusetts General Hospital, on “The “SPOT SMA” Longitudinal Population Database Repository (LPDR): a tool for the SMA community and their physicians” 
  14. Christine DiDonato PhD from Ann & Robert H. Lurie Children’s Hospital/Northwestern University on “Bridging Basic Science to Clinical Research and Outcomes: The Lurie Children’s Hospital experience”. 
  15. Elizabeth Luebbe MS from University of Rochester on “The SMA-Health Index: A Valid Instrument to Measure Therapeutic Benefit in Clinical Trials”.
  16. Stacy Rudnicki MD from Cytokinetics on “Update of CY 5021: A phase 2 clinical trial of reldesemtiv, a fast skeletal muscle troponin activator (FSTA), for the potential treatment of spinal muscular atrophy”.
  17. Hikari Tanaka from Uniformed Services University of the Health Sciences on “Protein stabilizer enhances ASO to improve SMA phenotype”.
  18. Meaghan Van Alstyne from Columbia University on “Mechanisms of Motor Neuron Death in SMA”.
  19. Oliver Gruss PhD and Utz Fischer PhD from Rheinische Friedrich-Wilhelms-Universität Bonn and University of Wuerzburg, Germany on “Identification of cellular signalling cues regulating the SMN complex and its implications for SMA”.
  20. Robin Parks PhD from the University of Ottawa on “Can tiny bits of cells in the blood help track the progress of SMA?”.
  21. Brunhilde Wirth PhD from the University of Cologne, Germany on “Genetic modifiers of SMA help some people to remain asymptomatic; How can this knowledge help all people with SMA?”.
  22. Erik Osman PhD from the University of Missouri on “Optimization of Morpholino Modified Anti-sense oligonucleotides”.
  23. Marc-Olivier Deguise from the University of Ottawa on “Metabolic considerations: Does fat intake matter in SMA?”.
  24. Allison Ebert PhD from the Medical College of Wisconsin on “Star Wars: How SMA astrocytes hurt motor neurons”.
  25. Jocelyn Côté PhD from the University of Ottawa on “SMN & Friends: What we can learn from identifying SMN interacting partners”.
  26. Arthur Burghes PhD and Corey Ruhno from Ohio State University on “Genetic variation affecting Spinal Muscular Atrophy severity”.
  27. Cera Hassinan from Johns Hopkins School of Medicine on “Impaired motor axon radial sorting and growth is followed by a rapid phase of axonal degeneration in severe SMA”.