Updating current knowledge on standards of care for SMA

In recognition of the changes in the care of patients with spinal muscular atrophy (SMA), it was felt that an update of standards of care (SOC) was needed. A new workshop that comprehensively reviewed the recent literature and took into account experts’ opinions on current standards of care has led to an updated consensus on standards of care in SMA.

The aims of this workshop were to:
i) examine each of the clinically meaningful topics of care for a patient with SMA and expand upon the number of areas of discussion from the initial 2007 effort;
ii) identify areas where consensus can be easily obtained based on striking evidence from published studies and to try to identify the best standards of care available. The relative degree of support and the estimated importance of an intervention would be developed for each recommendation;
iii) promote discussion among experts, identifying the areas of controversy that warrant further study and iv) include patients in this process and request comments from pharmaceutical companies with drugs under clinical development for SMA.
Finkel RS, Sejersen T, Mercuri E; ENMC SMA Workshop Study Group. 218th ENMC International Workshop:: Revisiting the consensus on standards of care in SMA Naarden, The Netherlands, 19-21 February 2016. Neuromuscul Disord. 2017 Jun;27(6):596-605.