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Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3 — EMOTAS  

Spinal Muscular Atrophy Type 3 Clinical Trial

Official title:
Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3.

Clinical Trial Summary

The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ).

Clinical Trial Description

Spinal muscular atrophy (SMA) is the second neuromuscular disease meet in children. SMA is a genetically transmitted disease inducing muscular weakness predominating on shoulders and hips. Currently, there is no effective therapy to slow the progression of the disease. SMA is due to a neuron motor attempt of the spinal cord and recently it has been demonstrated a neuromuscular junction (NMJ) involvement, according to recent studies. EMOTAS study aim to understand if NMJ abnormalities could have an impact on motor performance and fatigue in SMA type 3 ambulatory patients by electromyogram and to improve by non-invasive therapy quality of life of patients. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02227823
Study type Interventional
Source Centre Hospitalier Régional de la Citadelle
Contact
Status Completed
Phase Phase 2
Start date July 2014
Completion date July 2017
View Details
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