UK SMA Patient Registry

Page last updated: August 2026

The Registry is vitally important for the SMA Community. It collects medical and quality of life information directly from individuals who have SMA. This includes Patient Reported Outcome Measures (PROMS) > information. This information is essential for clinicians and researchers so that they can learn more about SMA and the impact of treatments and other interventions.

The information collected through the registry contributed to the National Institute for Health and Care Excellence’s (NICE) review and approval of funding for the drug treatments nusinersen and risdiplam.

The registry is vital for ensuring that patients’ voices and views about their own condition continue to directly contribute to research

How to Register

To register, you or your child should:

be a resident of the UK and Ireland
have a genetic test result that confirms a diagnosis of SMA, or
have a genetic test arranged in the near future.

Make your voice heard and find out more….

Register online >

How is the Registry funded and managed?

SMA UK was pleased to fund this important ‘patient-facing’ database over many years until, in 2020 the pharmaceutical industry took over this supporting role.

Coordinated from the John Walton Muscular Dystrophy Research Centre at Newcastle University. SMA UK joins clinicians, Muscular Dystrophy UK and TreatSMA as part of the patient registry steering committee.

Meet Aleks, the Patient Registry Curator

Hello! I’m Aleks Carver, and I have been the Project Manager of the UK SMA Patient Registry since April 2026.

I work in the John Walton Muscular Dystrophy Research Centre at Newcastle University. The UK SMA Patient Registry is a Newcastle University research project. It works closely with the national clinical research studies Adult SMA REACH and SMA REACH UK (children) and is an active member of the global TREAT-NMD Registry Network. I also manage the UK Myotonic Dystrophy and FSHD Patient Registries.

I am responsible for the day to day running of the registry and am therefore your first point of contact for any questions or comments. It’s always good to hear feedback from registry participants so please don’t hesitate to get in touch if needed (contact details below).

It’s also my job to make sure all of the data in the registry is complete, accurate and up to date. We collect information through questionnaires called patient-reported outcome measures (PROMs). These short questionnaires collect information from patients about their experience of daily life, their activities and quality of life. In addition to PROMs, the registry collects the TREAT-NMD SMA Expanded Core Dataset. If you haven’t updated your details for a while, or if there is any information missing from your registration, you will probably be hearing from me soon!

Aleks Carver
UK SMA Patient Registry – Project Manager
John Walton Muscular Dystrophy Research Centre
Translational and Clinical Research Institute
Newcastle University
International Centre for Life
Newcastle upon Tyne
NE1 3BZ

E: smaregistry@newcastle.ac.uk

E: aleks.carver@newcastle.ac.uk

T: 0191 241 8640