UK SMA Patient Registry activities

Standards of Care in the UK

In 2007 an International Standards of Care Consensus was reached for children, young people and adults with SMA. This was published and has since been available to medical teams and people affected by SMA and their families. In February 2016, clinical researchers and patient representatives from Europe and US met in Naarden, the Netherlands, to work on a major review of the International Standards of Care for SMA. A lay summary of this meeting is available on the workshop organiser’s website here.  

It was important that the reviewers understood the day-to-day care actually experienced by individuals with SMA so that they could use this information to inform their creation of new standards. At the beginning of 2016, the registry sent out a survey to registry participants in which individuals with SMA and their families were asked about their experiences of care in the UK. Some of the initial findings from the survey were presented at the meeting in February. The full results are published here

TREAT-NMD published ‘A Guide to the 2017 International Standards of Care for SMA’ 


This guide is for individuals living with 5q SMA, as well as their families/caregivers, in the hope to provide more information alongside the services of your healthcare provider. It highlights standards of care, what care you might expect and is hoped to aid with starting active discussions with medical teams surrounding your condition management and other available options.



The Adult SMA REACH website is now up and running, it explains all about this UK SMA project - how the data is integrated, the team involved and has a map with all the participating sites.


NICE (National Institute for Health & Care Excellence)

NICE's aim is to improve outcomes for people using public health and care services, like the NHS. In order for treatments to be standardised within the NHS, evidence-based research is required. NICE help produce this evidence-based guidance, ensure quality standards are met and developed, as well as providing information to practitioners and managers across the services. Cost implication and effectiveness of new drugs has an impact on whether a drug is recommended.