NCT05931211

Motor Function and Activity of Daily Life in Spinal Muscular Atrophy

Translation of Motor Function to Daily Activity Performance in Individuals With SMA

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 100 (estimated)
• Sponsor: National Taiwan University Hospital · Academic / Other
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities.

Detailed description

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease that causes progressive muscle weakness and atrophy and eventually leads to loss of function. Despite recent advances in disease-modifying treatments, SMA can still significantly impact an individual's functioning in daily living and quality of life. This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities.

Conditions

• Spinal Muscular Atrophy

Timeline

• Start date: 2023-06-30
• Primary completion: 2024-06-30
• Completion: 2024-12-31
• First posted: 2023-07-05
• Last updated: 2023-07-07

Locations

1 site across 1 country: Taiwan