The United Kingdom Spinal Muscular Atrophy Patient Registry

The UK SMA Patient Registry is a registry for patients in the United Kingdom and Ireland with Spinal Muscular Atrophy, coordinated by the John Walton Muscular Dystrophy Research Centre at Newcastle University. It collects patient-reported outcome measures through online questionnaires, alongside medical information, with patient consent, to supplement data from clinical studies and inform regulatory authorities, clinicians, researchers and pharmaceutical companies about the impact and effectiveness of treatments, and participants register online or by requesting a paper registration form.