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Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future.


ABSTRACT: Allogenic hematopoietic stem cell transplantation (HSCT) has proven to be a viable treatment option for a selected group of patients with mucopolysaccharidoses (MPS), including those with MPS types I, II, IVA, VI, and VII. Early diagnosis and timely referral to an expert in MPS are critical, followed by a complete examination and evaluation by a multidisciplinary team, including a transplantation physician. Treatment recommendations for MPS are based on multiple biological, sociological, and financial factors, including type of MPS, clinical severity, prognosis, present clinical signs and symptoms (disease stage), age at onset, rate of progression, family factors and expectations, financial burden, feasibility, availability, risks and benefits of available therapies such as HSCT, enzyme re

SUBMITTER: Taylor M 

PROVIDER: S-EPMC6615945 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature