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