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Unmet Cardiac Clinical Needs in Adult Mucopolysaccharidoses

The Mucopolysaccharidoses (MPSs) are a group of heterogenous disorders with complex multisystemic presentations. Although Haematopoietic Cell Transplantation (HCT) and Enzyme Replacement Therapy (ERT) have extended the lifespan of individuals affected with MPS well into adulthood, reversal of pre-ex...

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Detalles Bibliográficos
Autores principales: Stepien, Karolina M., Braunlin, Elizabeth A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9226406/
https://www.ncbi.nlm.nih.gov/pubmed/35757333
http://dx.doi.org/10.3389/fcvm.2022.907175