Protocol for a phase II, monocentre, double-blind, placebo-controlled, cross-over trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2–4 (SPACE trial)

INTRODUCTION: Hereditary proximal spinal muscular atrophy (SMA) is caused by homozygous loss of function of the survival motor neuron 1 gene. The main characteristic of SMA is degeneration of alpha motor neurons in the anterior horn of the spinal cord, but recent studies in animal models and patient...

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Bibliografiske detaljer
Udgivet i:BMJ Open
Main Authors: Stam, Marloes, Wadman, Renske I, Wijngaarde, Camiel A, Bartels, Bart, Asselman, Fay-Lynn, Otto, Louise A M, Goedee, H Stephan, Habets, Laura E, de Groot, Janke F, Schoenmakers, Marja A G C, Cuppen, Inge, van den Berg, Leonard H, van der Pol, W Ludo
Format: Artigo
Sprog:Inglês
Udgivet: BMJ Publishing Group 2018
Fag:
Neurology
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6067401/
https://ncbi.nlm.nih.gov/pubmed/30061431
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/bmjopen-2017-019932
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