An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)

Inclusion Criteria

• •1. Genetically confirmed SMA and number of SMN2 copies available
• •2. a. Patients identified by NBS and treated with disease modifying therapy (DMT)
• •(2)a,i 4 copies or more of SMN2 and not treated with DMT
• •(2)a,ii less than 4 copies of SMN2 and not treated with DMT
• •or
• •(2)b. Patients diagnosed due to a sibling or alternative means
• •(2)b,i 4 copies or more of SMN2 and not treated with DMT
• •(2)b,ii less than 4 copies of SMN2 and not treated with DMT
• •(3)Patients between 4 months and below 4 years at baseline. Inclusion of patients can be before 4 months of age
• •(4)Parent(s)/legal guardian(s) able to provide written informed consent prior to the patient's participation in the study
• •(5)Male or female
• •1. Typically developing child
• •2. Participant between 6 months and 4 years at inclusion
• •3. Parent(s)/legal guardian(s) able to provide written informed consent prior to the participation in the study
• •4. Male or female