Care Guides for Spinal Muscular Atrophy
2017 Standards 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, Spinraza ® (Nusinersen), was approved in the USA. Though this is a significant step it is acknowledged such treatments are not a cure. Treatments must be provided alongside the best possible supportive care and management in order to improve life expectancy and quality of life.
The first international guidelines to detail best standard of care (SOC) was published in 2007 entitled ‘Consenus statement for standards of care in spinal muscular atrophy’. You can read more about the development of these guidelines here (link to page ‘Previous care standards for SMA’).
The guidelines from 2007 were widely adopted by clinicians, patient representative and pharmaceutical companies. In 2016 twenty-six experts and patient representatives from nine countries convened in Naarden for a European Neuro Muscular Centre (ENMC) International workshop2. Experts agreed that although many aspects of care had dramatically improved , there was also new evidence to suggest improved natural history in all types of SMA due to advancements and more proactive approach in medical care. Considering this, and the rise of clinical trials, it was decided an update of the 2007 consensus statement was required.
Following the ENMC workshop the multidisciplinary committee of international experts worked to provide a two part updated Standards of care, the work was led by Professors Richard Finkel (USA) and Eugenio Mercuri (Europe) and published in neuromuscular disorders in 2017. Fifteen core group members worked with over one hundred experts through conference calls, e-mail communications meetings and Delphi survey. 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:
The Guide to the 2017 International Standards of Care for SMA
Clinicians, academics, health care professionals and patient representatives from Spinal Muscular Atrophy UK, Cure SMA, SMA Europe, Muscular Dystrophy UK and TREAT-NMD have used the 2017 care recommendations, to work together to produce a current and comprehensive medical guide aimed at people with SMA and their families.
We are sure the community will find it a valuable resource to ensure the very latest in medical care standards are accessible to all people living with SMA.
The guide has currently been translated into 10 different languages:
We would like to extend a very gracious thank you to the authors of the scientific articles for peer reviewing the content and to the target audience reviewers for your extensive feedback.
If you are able to help to translate the guide into your language please get in touch: info@treat-nmd.com
