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10 积分 2024-08-13 加入
Description of clinical and genetic features of 122 patients included in the Spanish Pompe registry
19小时前
求助中
Motor outcomes in patients with infantile and juvenile Pompe disease: Lessons from neurophysiological findings
21小时前
求助中
Long‐term follow‐up of 64 children with classical infantile‐onset Pompe disease since 2004: A French real‐life observational study
23小时前
已完结
Trends in the development of cellular and gene therapy in China
23小时前
已完结
Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium
1天前
已完结
Reduction of Preexisting AAV9 Antibody Titers Before Onasemnogene Abeparvovec Administration in Twins With Spinal Muscular Atrophy
5天前
已完结
China’s first approved gene therapy for hemophilia B: A new era for global AAV-based treatments
1个月前
已关闭
Diagnosis and managements of X - linked adrenoleukodystrophy
2个月前
已完结
Approval and therapeutic value of gene therapies in the US and Europe
2个月前
已完结
Optimality score for the neurologic examination of the infant at 12 and 18 months of age
3个月前
已完结
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