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

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 LSDs, inclu...

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Detalles Bibliográficos
Autores principales: Mehta, Atul, Ramaswami, Uma, Muenzer, Joseph, Giugliani, Roberto, Ullrich, Kurt, Collin-Histed, Tanya, Panahloo, Zoya, Wellhoefer, Hartmann, Frader, Joel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7788852/
https://www.ncbi.nlm.nih.gov/pubmed/33407729
http://dx.doi.org/10.1186/s13023-020-01645-9

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7788852/
https://www.ncbi.nlm.nih.gov/pubmed/33407729
http://dx.doi.org/10.1186/s13023-020-01645-9

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