Lv3
270 积分 2026-03-06 加入
Reduction of Preexisting AAV9 Antibody Titers Before Onasemnogene Abeparvovec Administration in Twins With Spinal Muscular Atrophy
7小时前
待确认
Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial
4天前
已完结
Plasma and cerebrospinal fluid neurochemical pattern in Menkes disease
25天前
已完结
Development of a new biochemical test to diagnose and monitor neuroblastoma in Vietnam: Homovanillic and vanillylmandelic acid by gas chromatography–mass spectrometry
29天前
已完结
Screening for Menkes disease using the urine HVA/VMA ratio
29天前
已完结
Plasma Catechols in Familial Dysautonomia: A Long-term Follow-up Study
29天前
已完结
Safety of AAV gene therapy programs for neurological indications – Learning from programs in non-lysosomal disorders
1个月前
已完结
The Griffiths Development Scales‐Chinese (GDS‐C): A cross‐cultural comparison of developmental trajectories between Chinese and British children
1个月前
已完结
Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery
1个月前
已完结
Epilepsy in Menkes disease: An electroclinical long-term study of 28 patients
1个月前
已完结
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