共 50 条
- [41] ‘Bespoke Gene Therapy Consortium’ sets out to enable gene therapies for ultra-rare diseases Nature Reviews Drug Discovery, 2021, 20 : 886 - 887
- [42] Ultra-rare renal diseases diagnosed with whole-exome sequencing: Utility in diagnosis and management BMC Medical Genomics, 14
- [43] Plasma fractionation unused intermediates proteome as a potential source of new therapies for ultra-rare diseases FEBS OPEN BIO, 2024, 14 : 122 - 122
- [45] Ultra-Rare Disorder in a Young Girl with Lipodystrophy: Analbuminemia INDIAN JOURNAL OF PEDIATRICS, 2021, 88 (07): : 723 - 723
- [47] Complement Turning Violent in Ultra-Rare Genetic Disorder JOURNAL OF IMMUNOLOGY, 2023, 210 (01):
- [48] Ultra-Rare Disorder in a Young Girl with Lipodystrophy: Analbuminemia Indian Journal of Pediatrics, 2021, 88 : 723 - 723