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Disease Characteristics, Effectiveness, and Safety of Vestronidase Alfa for the Treatment of Patients with Mucopolysaccharidosis Vii in a Novel, Longitudinal, Multicenter Disease Monitoring Program

AuthID
P-010-CXH

Author(s)

Giugliani, R

Gonzalez Meneses, A

Scarpa, M

Burton, B

Wang, R

Martins, E

Oussoren, E

Hennermann, JB

Chabrol, B

Grant, CL

Sun, A

Durand, C

Hetzer, J

Malkus, B

Marsden, D

Merritt, JL II

Document Type

Article

Year published

2024

Published

in ORPHANET JOURNAL OF RARE DISEASES

Volume: 19, Issue: 1

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Scopus^®

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Pubmed^®

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Metadata
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Publication Identifiers

DOI: 10.1186/s13023-024-03176-z

Pubmed: 38715031

SCOPUS: 2-s2.0-85192269332

Wos: WOS:001215641200002

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Disease Characteristics, Effectiveness, and Safety of Vestronidase Alfa for the Treatment of Patients with Mucopolysaccharidosis Vii in a Novel, Longitudinal, Multicenter Disease Monitoring Program

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Disease Characteristics, Effectiveness, and Safety of Vestronidase Alfa for the Treatment of Patients with Mucopolysaccharidosis Vii in a Novel, Longitudinal, Multicenter Disease Monitoring Program

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