A charitable access program for patients with lysosomal storage disorders in underserved communities worldwide

Abstract Background Lysosomal storage disorders (LSDs) are rare genetic disorders, with heterogeneous clinical manifestations and severity. Treatment options, such as enzyme replacement therapy (ERT), substrate replacement therapy, and pharmacological chaperone therapy, are available for several LSD...

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Principais autores:	Atul Mehta, Uma Ramaswami, Joseph Muenzer, Roberto Giugliani, Kurt Ullrich, Tanya Collin-Histed, Zoya Panahloo, Hartmann Wellhoefer, Joel Frader
格式:	Artigo
語言:	Inglês
出版:	BMC 2021-01-01
叢編:	Orphanet Journal of Rare Diseases
主題:	Gaucher disease Fabry disease Hunter syndrome MPS II Access Lysosomal storage disorders
在線閱讀:	https://doi.org/10.1186/s13023-020-01645-9
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