Cure SMA Funded Standard of Care Paper Published in Neuromuscular Disorders Journal

The journal Neuromuscular Disorders has published a paper reporting on the findings of an international workgroup convened to update the Standard of Care (SOC) for SMA. Cure SMA provided funding support for this project, and Mary Schroth, medical director for Cure SMA, served on the workgroup.

A SOC for SMA was first issued in 2007, following the 2005 establishment of a workgroup to study the topic. The current workgroup was charged with re-evaluating this document in light of the changes that have taken place in SMA treatment and care over the last decade.

With the approval of Spinraza and the continued progress of other programs in the SMA drug pipeline, the issue of care is more important that ever. Access to treatments must be combined with improved care to both extend lifespan and enhance quality of life for those affected by SMA.

The paper’s authors also cited the progress in both care and treatment for SMA as part of the impetus for this workgroup. “Over the last decade many aspects of care for infants and children with SMA have dramatically improved, resulting in longer survival and better quality of life,” they write. “Several papers have reported a large amount of data reflecting improvements in care in orthopedic management of scoliosis, nutrition, respiratory support and other aspects. In recognition of these changes in the care of patients with SMA, it was felt that an update of SOC was needed.”

The workgroup met in The Netherlands in February 2016 to discuss clinically meaningful topics of care for SMA. Whereas just five topics were covered in the 2005 workgroup (diagnostics and new interventions, pulmonary care, nutritional issues, orthopedic care and rehabilitation, and palliative care), this new workgroup covered nine topics. Orthopedics and rehabilitation were considered separately, and other organ system involvement, acute care in the hospital, and medication were added.

The goal was to identify areas where new consensus has emerged from recent studies, and articulate that consensus. The workgroup also identified areas where further study is still required. Finally, the group surveyed patients and families to incorporate their voice into this process.

The workgroup also identified several key next steps for continued improvement to the SMA SOC:

  • A separate workshop to discuss and evaluate medications commonly used in the treatment and management of SMA. 
  • Additional sessions to cover topics not addressed by the nine primary areas named above. This includes dental care, pregnancy in SMA, and sexuality.
  • Further work and research in areas, such as nutrition and palliative care, in which a consensus has not yet been reached.

Our thanks to the members of the workgroup for their dedication and expertise on this project.

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