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

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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Bibliographic Details
Main Authors: Atul Mehta, Uma Ramaswami, Joseph Muenzer, Roberto Giugliani, Kurt Ullrich, Tanya Collin-Histed, Zoya Panahloo, Hartmann Wellhoefer, Joel Frader
Format: Article
Language:English
Published: BMC 2021-01-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Gaucher disease
Fabry disease
Hunter syndrome
MPS II
Access
Lysosomal storage disorders
Online Access:https://doi.org/10.1186/s13023-020-01645-9

Internet

https://doi.org/10.1186/s13023-020-01645-9

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