UK SMA Patient Registry

Spinal Muscular Atrophy UK funds the UK SMA patient registry.

 

Meet Lindsay,
the Patient Registry Curator 

 

Read the FAQs below to find out more about the registry and how SMA UK is involved:

What is a patient registry for SMA?

A database of genetic and clinical information (or data) about people affected by SMA.

How is the information used?

Promising new treatments for SMA are becoming available. More treatments are in development and  need to be tested in clinical trials. However, SMA is a rare condition and it can sometimes take years to find enough patients for a clinical trial. The Patient Registry can help speed this up. The Registry also helps specialists gain more knowledge about the condition and the number of people affected by SMA. In addition, it helps capture information from individuals now receiving treatment. This information helps to develop and improve worldwide standards of care for people with SMA.

What information is collected?

Personal details, such as name, address, date of birth and gender, so that individuals can be identified and contacted.
Clinical and genetic information to inform researchers in their development of treatments, to aid recruitment to clinical trials and to assist access to new treatments.

What other benefits are there?

If you register, you will be told about clinical trials and other studies you may wish to join, but you are not obliged to do so. You will receive information relevant to SMA about latest research developments and about TREAT-NMD activities. Collected information will help improve standards of care for people with SMA and will help inform on the impact of new therapies.

Is my data safe?

All the information is stored in a secure server which is protected in a similar way to online bank accounts. Only specially appointed registry staff can look at your information. The UK SMA Patient Registry is part of the TREAT-NMD Global SMA Registry, which collects medical information from national SMA registries worldwide. Only anonymised data is shared with the TREAT-NMD Global SMA registry. You can be sure that your contact details are safe.

The UK SMA Patient Registry also collaborates with SMA REACH UK, a research database that collects longitudinal clinical data from children with SMA. SMA REACH UK collects patient data entered by doctors and is operated by Great Ormond Street Hospital and the MRC Neuromuscular Centres in London, Newcastle and other neuromuscular centres across the country. This compliments data provided by patients themselves in the UK SMA Patient Registry. Consent for the exchange of limited and specific patient data between the registry and database is requested at registration.

How is Spinal Muscular Atrophy UK involved with the UK SMA Patient Registry?

Spinal Muscular Atrophy UK funds the UK SMA Patient Registry. By doing so, it is supporting clinical research and ensuring all people with SMA in the UK and in Ireland can be informed about research, clinical trials and best-practice care. Information you provide to the UK SMA Patient Registry is not shared with SMA UK.

Who should register?

Adults and children with SMA and a confirmed mutation in the SMN1 gene who live in the UK or Ireland. SMA is a rare condition, every single person counts!

How do I register?

You can register online and create an account so that you can view and update your information at any time. If a person is under 16, a parent or guardian much create an account on their behalf. 

Registration is voluntary and at all times the information remains your property. You have the right to withdraw your information at any time.