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Ethical issues in managing Lysosomal storage disorders in children in low and middle income countries
The lysosomal storage diseases are a group of rare, inherited metabolic diseases affecting about 1 in 7000 to 8000 people. In recent years, the introduction of enzyme replacement therapy, substrate reduction therapy and small molecule therapy, has changed the natural course of this otherwise progres...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Professional Medical Publications
2017
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5648935/ https://www.ncbi.nlm.nih.gov/pubmed/29067088 http://dx.doi.org/10.12669/pjms.334.12975 |