Unmet Cardiac Clinical Needs in Adult Mucopolysaccharidoses

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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Bibliographic Details
Main Authors: Karolina M. Stepien, Elizabeth A. Braunlin
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-06-01
Series:Frontiers in Cardiovascular Medicine
Subjects:
adult mucopolysaccharidoses
cardiac
unmet needs
therapies
transition
Online Access:https://www.frontiersin.org/articles/10.3389/fcvm.2022.907175/full

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