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IKZF1 and UBR4 gene variants drive autoimmunity and TH2 polarization in IgG4-related disease
Download from doi.orgEfficacy and Safety of Taliglucerase Alfa for the Treatment of Gaucher Disease: A 9-Year Experience
Download from doi.orgEarly role for a Na + ,K + -ATPase ( ATP1A3 ) in brain development
Download from doi.orgEstimated prevalence of mucopolysaccharidoses from population-based exomes and genomes
Download from doi.orgPrecision Medicine for Lysosomal Disorders
Download from doi.orgLoss of the sphingolipid desaturase DEGS1 causes hypomyelinating leukodystrophy
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