• General Spinal Muscular Atrophy (SMA)
  • Nusinersen
    • The following areas of research are currently being considered with a focus on teen-adult population.

      Clinical effectiveness

      • Longitudinal effectiveness and safety of treatment with nusinersen
      • Characterization of new patient phenotypes or trajectories on therapy with nusinersen
      • Effect of nusinersen on other domains (i.e. bulbar function, cognition, respiratory function, etc.)

      Treatment and management strategies

      • Complex spines: administration, surgical techniques, etc.
      • Evolving standards of care

      Patient experience and health economics

      • Health care resource utilization & burden of illness
      • Quality of life measures and outcomes


      • Identification and validation of biomarkers of disease progression or response to nusinersen therapy in SMA

      Research support is awarded on a highly competitive basis, and submission of research proposals in these areas of interest does not guarantee that support will be awarded.