Adult SMA REACH: Standardised Data Collection Study in UK Adults with Spinal Muscular Atrophy

What this Trial Is About

Researchers are studying adults with Spinal Muscular Atrophy (SMA) to better understand how current care standards and new treatments affect the natural course of this genetic motor neuron disease. This observational study builds on previous efforts that gathered data from children with SMA and collaborates with several national and international SMA networks to improve patient care and support future research. The study is sponsored by Newcastle upon Tyne Hospitals NHS Foundation Trust and funded by Biogen and Roche. Participants are adults aged 16 and older with a genetically confirmed diagnosis of 5q SMA. The study collects data on patients receiving available SMA treatments such as Risdiplam and Nusinersen. There is no intervention or placebo; instead, the study monitors and records patient outcomes over time to learn more about the disease and its management. During the study, participants undergo assessments every six months using several measures including the World Health Organization Motor Milestones, Revised Upper Limb Module, 6 Minute Walk Test, Hammersmith Functional Motor Scale Expanded, and the Egen classification 2 scale. These evaluations track motor function and disease progression. The study involves regular data collection from treatment initiation through study completion, helping researchers gather important long-term information about adult SMA.

Adult SMA Research and Clinical Hub