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60 积分 2023-10-01 加入
Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial
27天前
已完结
Semiquantitative Scale for Assessing Brain MRI Abnormalities in Wilson Disease: A Validation Study
27天前
已完结
Neurofilaments as biomarkers in neurological disorders - towards clinical application
27天前
已完结
Serum Neurofilament Light Chain as a Biomarker of Brain Injury in Wilson's Disease: Clinical and Neuroradiological Correlations
27天前
已完结
AAV gene therapy for autosomal recessive deafness 9: a single-arm trial
27天前
已完结
In vivo base editing gene therapy for heterozygous familialhypercholesterolemia:a phaseItrial
1个月前
已完结
AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial
1个月前
已完结
Clinical consequences of interrupting enzyme replacement therapy in children with type 1 Gaucher disease
2个月前
已完结
In vivo chimeric antigen receptor (CAR)-T cell therapy
2个月前
已完结
Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1
2个月前
已关闭
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