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Rare DiseasesRare DiseaseEnzyme Replacement TherapyFabry DiseaseNewborn ScreeningDuchenne Muscular DystrophyGene TherapyFamilial HypercholesterolemiaGenomic SequencingMedicines DevelopmentClinical TrialsRare Disease PatientsGenetic DiseaseOrphan DrugsRare Genetic DiseasesOrphan DrugLysosomal Storage DisordersSystemic Lupus ErythematosusPrader-Willi SyndromeSpinal Muscular Atrophy
vol.4 (2025)
vol.3 (2024)
vol.2 (2023)