Bone and adrenal health in children with spinal muscular atrophy

This study uses wearable devices (like smartwatches or activity trackers) to monitor movement and motor development in young children with spinal muscular atrophy (SMA), a rare genetic condition affecting muscle strength. The goal is to see if wearables can track disease progression and treatment response better than traditional clinic visits.

This trial is a registry that collects health information from people with spinal muscular atrophy (SMA). It helps researchers understand the condition better and may support future studies and treatments.

This study looks at how people with spinal muscular atrophy (a genetic condition that affects muscle strength) experience changes in school – like moving from primary to secondary school or starting college. If you take part, you'll answer a questionnaire about your experiences, which may help schools and support services improve how they help others.

This study is looking for children and teens with spinal muscular atrophy (SMA) who are already on a treatment like Spinraza, Evrysdi, or Zolgensma. The goal is to understand different forms of SMA and how the treatment affects them over time.

This study collects health information from people with spinal muscular atrophy (SMA) over time to learn more about the disease and how to improve care. It may help researchers understand SMA better.

This study looks at how muscles work in children and young adults with spinal muscular atrophy (SMA) who are taking or starting SMN-directed therapies (like Evrysdi, Spinraza, or Zolgensma). It aims to understand the body's response to these treatments, which could help improve future care.