Rare Diseases

  • General Spinal Muscular Atrophy (SMA)

    • The following areas of research are currently being considered to support studies related to Spinal Muscular Atrophy (SMA).

      General SMA

      • Natural history of teens and adults with SMA
      • Newborn screening

      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.

  • Nusinersen