| 项目编号 |
PRJCA020590 |
| 项目标题 |
A Phase 4, open label, safety and efficacy study of Fabrazyme (agalsidase beta) as enzyme replacement therapy in Chinese participants with Fabry disease |
| 涉及领域 |
Medical |
| 数据类型 |
Whole genome sequencing
Variation
|
| 物种名称 |
Homo sapiens
|
| 描述信息 |
Based on the Phase 3 study and its extension study as well as the Phase 4 study results, it was demonstrated that early treatment may alleviate symptoms and prevent further progression of this lethal disease before irreversible damage has occurred. |
| 样品范围 |
Multiisolate |
| 发布日期 |
2023-10-18 |
| 出版信息 |
| PubMed ID |
文章标题 |
杂志名称 |
Doi |
发表年份 |
| 40760696
|
A phase 4, open-label, multicenter study of the safety and efficacy of agalsidase beta in Chinese patients with Fabry disease
|
Orphanet Journal of Rare Diseases
|
10.1186/s13023-025-03950-7
|
2025
|
|
|
| 项目资金来源 |
| 机构 |
项目类型 |
授权项目ID |
授权项目名称 |
| Other
|
Other
|
LPS16583
|
A Phase 4, open label, safety and efficacy study of Fabrazyme (agalsidase beta) as enzyme replacement therapy in Chinese participants with Fabry disease
|
|
|
| 提交者 |
Nan
Chen (cnrj100@126.com)
|
| 提交单位 |
Ruijin Hospital, Shanghai jiaotong University School of Medicine |
| 提交日期 |
2023-10-18 |
