Listar Ciencias de la Salud por tema "Humanitarian"
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A charitable access program for patients with lysosomal storage disorders in underserved communities worldwide
(2021) [Artículo de periódico]Background: Lysosomal storage disorders (LSDs) are rare genetic disorders, with heterogeneous clinical manifesta‑ tions and severity. Treatment options, such as enzyme replacement therapy (ERT), substrate replacement ...