Cargando…
Long-term follow-up and sudden unexpected death in Gaucher disease type 3 in Egypt
OBJECTIVE: To describe the long-term follow-up and distinct phenotype of a large cohort of patients with Gaucher disease type 3 on enzyme replacement therapy (ERT) in Egypt. METHODS: A prospective cohort study of 78 patients on ERT who were followed for up to 9 years with yearly evaluations that inc...
| Autores principales: | , , |
|---|---|
| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Wolters Kluwer
2016
|
| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4830203/ https://www.ncbi.nlm.nih.gov/pubmed/27123474 http://dx.doi.org/10.1212/NXG.0000000000000055 |