We are delighted to announce changes in the UK SMA Patient Registry! These developments will see the collection of information from adults and parents of children and young people living with SMA about their experience of daily life, their activities and quality of life. Also known as patient-reported outcome measures or PROMs, this information will be captured in short online questionnaires in the patient registry.
In the UK, many individuals are now able to receive SMA treatment. The perspective and observations of people living with this condition, about the impact it has on them and how any treatment may change this, is increasingly recognised. This is the drive behind the collection of this information. With patient consent, PROMs information from the patient registry will supplement data from the clinical studies SMA REACH UK (children) and Adult SMA REACH. It will help inform regulatory authorities, clinicians, researchers and pharmaceutical companies about the impact and effectiveness of treatments. It will be important information when regulatory authorities are deciding whether NHS funding of a treatment should continue.
Any parent of a child living with SMA or adult aged 16 years or over can register themselves in the UK SMA Patient Registry at https://www.sma-registry.org.uk/. All participants are encouraged to report their PROMs, in addition to the medical information also collected by the patient registry.