Study on the disease burden of patients with mucopolysaccharidosis type II in China
This study found that patients with MPS II were difficult to diagnose and easily misdiagnosed, their physical functions were impaired in many aspects. The existing treatment options are not sufficient in terms of economy and effectiveness, and there is also a lack of corresponding policy guarantees and support, which makes patients and their families have to face huge financial pressure.
Authors
- H Chen
- Min Li (ORCID: https://orcid.org/0000-0003-1330-2326)
- Ji Luo (ORCID: https://orcid.org/0000-0001-5504-3129)
- Hongjie Yu (ORCID: https://orcid.org/0000-0002-6335-5648)
- Fengxin Dong (ORCID: https://orcid.org/0000-0002-4454-2907)
- Shanshan Wang (ORCID: https://orcid.org/0000-0002-7205-3844)
- Shengnan Duan
- Yuan Ni (ORCID: https://orcid.org/0000-0002-9672-3531)
- Ya-Qun Wang
Institutions
- Jiamusi University (CN)
- Dalian Medical University (CN)
- First Hospital of China Medical University (CN)
- China Medical University (CN)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2024-11-05
- DOI
- https://doi.org/10.1186/s13023-024-03432-2
- Citations
- 3
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.98