Effect of Ambroxol chaperone therapy on Glucosylsphingosine (Lyso-Gb1) levels in two Canadian patients with type 3 Gaucher disease

Type 3 Gaucher disease (GD3) is characterized by progressive neurological features in addition to the typical systemic manifestations. Enzyme replacement therapy (ERT), the main stay treatment for Gaucher disease (GD), is not efficacious for the neurological manifestations. Ambroxol, in combination...

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Dades bibliogràfiques
Publicat a:Mol Genet Metab Rep
Autors principals: Charkhand, Behshad, Scantlebury, Morris H., Narita, Aya, Zimran, Ari, Al-Hertani, Walla
Format: Artigo
Idioma:Inglês
Publicat: Elsevier 2019
Matèries:
Short Communication
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6713848/
https://ncbi.nlm.nih.gov/pubmed/31467847
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2019.100476
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