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Clinical Trial Summary

The goal of this study is to investigate the acceptability, feasibility, safety and efficacy of an optimized rehabilitation program for treated patients with spinal muscular atrophy (SMA) compared to the current rehabilitation program in the United Kingdom. The aim is to provide patients with more hands on physiotherapy and access to rehabilitation devices at home to support parents currently providing rehabilitation on their own.


Clinical Trial Description

The main study objective is to investigate whether an optimized rehabilitation program would be accepted and is feasible within the SMA patient community. The optimized program will comprise of goal orientated rehabilitation with more frequent hands on physiotherapy (every 2 weeks) as well as a SMA approved rehabilitation device used at home on a weekly basis, to provide the most appropriate therapy for each individual patient. All study participants will receive the optimized rehabilitation program over 12 months. An external control group will be used to determine the potential efficacy. Research study visits will take place at baseline, month 6 and month 12. At these visits a general physical exam will be undertaken alongside collecting the participant and carer perception, satisfaction and compliancy of the rehabilitation program. Motor function assessments according to age and ambulatory status will be assessed to help evaluate potential therapeutic benefits. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT06419322
Study type Interventional
Source University of Oxford
Contact Serena Hill
Phone 01865618799
Email ace.sma@paediatrics.ox.ac.uk
Status Recruiting
Phase N/A
Start date June 2024
Completion date December 2026

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