2017 Standard of Care for Spinal Muscular Atrophy (SMA)

The clinical spectrum of Spinal Muscular Atrophy (SMA) means patients often require comprehensive, multi-disciplinary medical care. In December 2016, the first treatment for SMA, called nusinersen and marketed as SpinrazaTM, was approved in the USA. Though this is hugely positive, it is widely acknowledged such treatments are not a cure and must be provided alongside the best possible supportive care and management in order to achieve better quality of life and improve life expectancy of individuals with SMA.

The first international guidelines to detail best standard of care (SOC) was published in 2017. The guidelines which cover five areas of care (Diagnosis and management, pulmonary care, nutritional, orthopaedic and rehabilitation and palliative care) was published in an effort to address the differences in medical practice, increase healthcare providers knowledge and to set a baseline standard for future clinical trials. The guidelines were successful and widely adopted by clinicians, patient representative and pharmaceutical companies.

In 2016 twenty-six experts from nine countries and patient representatives convened in Naarden for a European Neuro Muscular Centre (ENMC) International workshop2. Experts agreed many aspects of care for infants and children with SMA have dramatically improved since the 2007 publication mostly, with respect to orthopaedic management, nutrition and respiratory support. There were also new aspects of care which were not included in the original article and evidence of improvement in the natural history of all the SMA types. Considering this it was therefore decided an update of the consensus statement was required.

Updated 2017 Standard of Care for SMA

Following the ENMC workshop the multidisciplinary committee of international experts worked to provide a two part update of the topics covered in the previous 2007 recommendations. The update addresses nine key areas of care, including new topics such as, acute and emergency care, medications and the involvement of other organs. You can access the updated care recommendations here:

Part 1: Diagnosis and Management of SMA; Recommendations for diagnosis; rehabilitation; orthopaedic and nutritional care.

Part 2: Diagnosis and Management of SMA; Pulmonary and acute care; medication; supplements and immunisations; other organ systems; and ethics.

TREAT-NMD are now working with multiple patient organisations to produce an updated SMA family guide. The guide is expected to be available by the end of 2018.


1. Wang CH, Finkel RS, Bertini ES, Schroth M, Simonds A, Wong B, Aloysius A, Morrison L, Main M, Crawford TO, Trela A Participants of the International Conference on SMA Standard of Care (2007) ‘Consensus statement for standard of care in spinal muscular atrophy’. Journal of Child Neurology, 22(8), pp.1027-49 doi.org/10.1177/0883073807305788

2. Finkle RS, Serjesen T, Mercuri E; ENMC SMA Workshop Study Group (2017) ‘218th ENMC International Workshop:Revisiting the consensus on standards of care in SMA Naarden, The Netherlands, 19-21 February 2016’. Neuromuscular Disorders. 27 pp. 596-605. doi.org/10.1016/j.nmd.2017.02.014.

18 Sep 2018