We are delighted to announce changes in the UK SMA Patient Registry! Information about the daily experience of those living with SMA can now be collected to gain insight into 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 people living with SMA are now able to receive treatment. And with this, the impact the condition has on those with SMA 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 especially important information when regulatory authorities are deciding whether NHS funding of a treatment should continue. If you’re a parent of a child living with SMA or an adult aged 16 years or over, you can register in the UK SMA Patient Registry.
All participants are encouraged to report their PROMs, in addition to the medical information also collected by the patient registry.