Overview of therapeutic approaches for spinal muscular atrophy

Information on current best practice in care for people with spinal muscular atrophy (SMA) is provided in the SMA care standards section of this website. This research overview section aims to inform patients and their families about the approaches currently being investigated for the possible future treatment of SMA – therapies that are currently still at the laboratory or clinical trial stage. It discusses the strengths and limitations of each approach. These factors need to be carefully assessed before the alternatives described here can be implemented clinically.

Many developments are taking place in the therapeutic approaches in SMA. This is a dynamic and promising field but as a detailed account of progress is outside the scope of this short review we provide a general overview of present investigation into SMA.