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How We Support Research And Access To Treatments

Access to New Treatments

We are committed to working with the SMA community to campaign for access to treatments that have been proven effective in clinical trials. Our current focus is nusinersen. Read more   

Our Research Correspondents

Dr James Sleigh, our Scientific Research Correspondent, and Dr Alex Murphy, our Clinical Care Research Correspondent, keep the SMA community up-to-date with latest news about treatment developments and management research. Keep informed by signing up for our mailings.

Input to Research

SMA researchers often ask our support services team for insights into the impact of SMA on people's daily lives. We also enable opportunities for people's own voices to be heard directly via surveys and consultations posted on our research noticeboard as well as in our monthly e-newsletter and on social media.

The UK SMA Patient Registry

We help to fund this important database that links children and adults who have SMA with researchers conducting studies including clinical trials of new drugs and treatments. Find out more.

International Standards of Care

These guide day-to-day care and practice for professionals and families. You can read about, and download, the current 2008 version here. In February 2016, we helped fund the start of their major review by international experts and patient representatives. We are now helping produce the new family-friendly version. 

International Links

We are active members and funders of SMA Europe, an umbrella organisation of patient and research groups. It is a vital information network that also funds and supports scientific and medical research. We also regularly attend the USA Cure SMA Conferences and follow other international SMA developments.

Rare Diseases Day

Rare Disease Day takes place annually on the last day of February. We always take part in an event to raise awareness of SMA. Read about what we did in 2017 here.  

Our Previous Direct Funding of Research Projects

Our funds and staff time currently focus on all the above endeavours. We are also proud to have made a large financial contribution to many projects that have enabled SMA research to reach this exciting point. You can see what projects we funded between                           1986 and 2014 here.