This is a single arm, prospective, open label, multicenter study in participants who are diagnosed with Gaucher disease type III
Title | This is a single arm, prospective, open label, multicenter study in participants who are diagnosed with Gaucher disease type III |
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Description | Approximately 12 participants will be assigned to study intervention in the study in order to yieldat least 10 evaluable patients at the end of 52 weeks. Patient drop-out rate will be monitored for the purpose of adjusting the sample size. |
Organism | Homo sapiens |
Data Type | Other Type of Genomic Data |
Data Accessibility | Open-access |
BioProject | PRJCA024236 |
Release Date | 2024-04-01 |
Submitter | zhengqing qiu (zhengqingqiu33@aliyun.com) |
Organization | Peking Union Medical College Hospital- Chinese Academy of Medical Sciences-Peking Union Medical College |
Submission Date | 2024-03-12 |
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File ID | File Title | Number/Samples | File Type | File Size | File Suffix | Download Times | Download |
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OMIX005974-01 | Genetic Data for 3939 Project | 1 | Other Type of Genomic Data | 10.1 KB | rar | 0 |