Promising Effect of High Dose Ambroxol Treatment on Neurocognition and Motor Development in a Patient With Neuropathic Gaucher Disease 2

Gaucher Disease (GD) 2 is a rare inherited lysosomal disorder. Early-onset and rapid progression of neurovisceral symptoms lead to fatal outcome in early childhood. Treatment is symptomatic, a curative therapy is currently not available. This prospective study describes the clinical and biochemical...

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Detalles Bibliográficos
Autores principales: Aries, Charlotte, Lohmöller, Benjamin, Tiede, Stephan, Täuber, Karolin, Hartmann, Guido, Rudolph, Cornelia, Muschol, Nicole
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Neurology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9207411/
https://www.ncbi.nlm.nih.gov/pubmed/35734474
http://dx.doi.org/10.3389/fneur.2022.907317

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