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60 积分 2024-11-14 加入
High prevalence of GAA c.[752C > T;761C > T] haplotype complicates high-risk screening for Pompe disease in the Chinese population
1天前
已完结
Glycogen storage diseases
3个月前
已完结
Successful desensitization to alglucosidase alfa in a very young infant with Pompe disease
5个月前
已关闭
AAV9-Mediated Gene Therapy for Infantile-Onset Pompe’s Disease
8个月前
已完结
Gene Therapy for Infantile-Onset Pompe’s Disease
8个月前
已完结
Biologics to treat anaphylaxis
8个月前
已完结
A rare homozygous mutation in the YARS2 gene presents with hypertrophic cardiomyopathy, lactic acidosis and anemia in a Chinese infant
9个月前
已完结
Long QT syndrome in children and adolescents: risk factors and outcomes in a large German cohort
10个月前
已关闭