Research Projects

Our ultimate goal is to find an effective treatment and cure for SMA. In order to achieve this, we fund projects and initiatives to address the following areas:

  • Basic research into the cause of SMA
  • SMA Models (an important tool for understanding the basic disease mechanisms and for testing potential therapies)
  • Gene therapy and DNA based therapies (to correct the basic defect in SMA and either prevent the disease developing if given early, or maintain and improve function when the disease is already established)
  • Other potential therapies for SMA (such as those that protect the motor neurons)
  • Outcome measures and biomarkers (measurements that can be used to assess disease progression or how patients respond to potential new treatments in clinical trials)

In our quest to achieve this, our research strategy has three distinct parts:

Find out more about our research portfolio:


Find out more about the research scientists who are heading up our projects.