Thema
Journal Article
3.388
Rare diseases
2.205
Rare disease
1.711
rare disease
1.459
rare diseases
1.418
Research Support, Non-U.S. Gov't
1.022
Review
863
Medicine
641
R
610
Rare Disease
374
Case Reports
357
Rare Diseases
282
Orphan drugs
226
Research Support, N.I.H., Extramural
196
Genetics
193
610
175
Quality of life
156
Medicine & Public Health
136
orphan drugs
135
Medicine (General)
134
Diagnosis
131
医药、卫生
125
医药卫生科技
125
Pediatrics
122
Epidemiology
117
Biology (General)
108
罕见病
104
Clinical trials
99
Study Type: Observational
99
COVID-19
98
Registry
87
orphan drug
86
precision medicine
85
Systematic Review
76
Children
75
Recruitment Status: Recruiting
75
diagnosis
74
Qualitative research
72
gene therapy
71
Orphan drug
70
Editorial
69
Public aspects of medicine
68
Genomics
67
Study Type: Interventional
67
Orphan Drug
65
Registries
62
genetics
61
China
60
Drug development
58
Precision medicine
58
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