Dose-dependent effects of enzyme replacement therapy on skeletal disease progression in mucopolysaccharidosis VII dogs

Dose-dependent effects of enzyme replacement therapy on skeletal disease progression in mucopolysaccharidosis VII dogs

Mucopolysaccharidosis (MPS) VII is an inherited lysosomal storage disorder characterized by deficient activity of the enzyme β-glucuronidase. Skeletal abnormalities are common in patients and result in diminished quality of life. Enzyme replacement therapy (ERT) for MPS VII using recombinant human β...

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Bibliographic Details
Main Authors: Rahul Gawri, Yian Khai Lau, Gloria Lin, Snehal S. Shetye, Chenghao Zhang, Zhirui Jiang, Khaled Abdoun, Carla R. Scanzello, Stephanie Y. Jo, Wilfried Mai, George R. Dodge, Margret L. Casal, Lachlan J. Smith
Format: Article
Language:English
Published: Elsevier 2023-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
mucopolysaccharidosis
Sly syndrome
spine
bone
synovial joint
enzyme replacement therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122001656

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http://www.sciencedirect.com/science/article/pii/S2329050122001656

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