共 50 条
- [48] Spinal muscular atrophy: A test case for drug development in orphan diseases NeuroRX, 2006, 3 (4): : 535 - 539
- [49] A methodological framework for drug development in rare diseases Orphanet Journal of Rare Diseases, 9
- [50] Regulatory Framework for Drug Development in Rare Diseases JOURNAL OF CLINICAL PHARMACOLOGY, 2022, 62 : S15 - S26