Safety and efficacy of laronidase in Chinese patients with mucopolysaccharidosis type I: a phase IV, single-arm, open-label, multicenter study

Authors

Chaochun Zou (ORCID: https://orcid.org/0000-0002-4667-3636)
Xiaoping Luo (ORCID: https://orcid.org/0000-0002-3748-4817)
Yan Liang (ORCID: https://orcid.org/0000-0003-4029-1765)
Yuqing Jiao (ORCID: https://orcid.org/0000-0003-2757-8948)
Yan-Ling Yang
Xin Liu (ORCID: https://orcid.org/0000-0003-4880-6321)
Li Liu (ORCID: https://orcid.org/0000-0002-0957-636X)
Yu Wang (ORCID: https://orcid.org/0000-0001-7959-3387)

Institutions

Peking University (CN)
Children's Hospital of Zhejiang University (CN)
Peking University First Hospital (CN)
Sanofi (China) (CN)
Boehringer Ingelheim (China) (CN)
Tongji Hospital (CN)
Huazhong University of Science and Technology (CN)
Hubei University (CN)
Guangzhou Medical University (CN)

Publication Details

Journal: Orphanet Journal of Rare Diseases
Published: 2025-10-29
DOI: https://doi.org/10.1186/s13023-025-04056-w
Primary Topic: Lysosomal Storage Disorders Research
Type: article
Field-Weighted Citation Impact: 0.00

Funders

Sanofi

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article

Safety and efficacy of laronidase in Chinese patients with mucopolysaccharidosis type I: a phase IV, single-arm, open-label, multicenter study

Chaochun Zou, Xiaoping Luo, Yan Liang, Yuqing Jiao et al.

Oct 29, 2025

Orphanet Journal of Rare Diseases

Lysosomal Storage Disorders Research

article

Safety and efficacy of laronidase in Chinese patients with mucopolysaccharidosis type I: a phase IV, single-arm, open-label, multicenter study

Chaochun Zou, Xiaoping Luo, Yan Liang, Yuqing Jiao, Yan-Ling Yang, Xin Liu, Li Liu, Yu Wang

October 29, 2025article en

Abstract

No abstract available for this paper.

Orphanet Journal of Rare DiseasesVol. 20(1)

Peking University (CN), Children's Hospital of Zhejiang University (CN), Peking University First Hospital (CN), Sanofi (China) (CN), Boehringer Ingelheim (China) (CN), Tongji Hospital (CN), Huazhong University of Science and Technology (CN), Hubei University (CN), Guangzhou Medical University (CN)

Sanofi

Openalex Percentile: Top 61%

Lysosomal Storage Disorders Research

DOI Open Access PDF OpenAlex

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Safety and efficacy of laronidase in Chinese patients with mucopolysaccharidosis type I: a phase IV, single-arm, open-label, multicenter study — Chaochun Zou, Xiaoping Luo, et al. · Orphanet Journal of Rare Diseases (2025) | TGRS Research Map | TGRS