共 50 条
- [46] NHS England agrees deal for gene therapy for spinal muscular atrophy BMJ-BRITISH MEDICAL JOURNAL, 2021, 372 : n653
- [47] Cost-effectiveness of screening newborns for spinal muscular atrophy GESUNDHEITSOEKONOMIE UND QUALITAETSMANAGEMENT, 2022, 27 (06): : 282 - 282
- [48] Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of Spinal and Bulbar Muscular Atrophy CELL REPORTS, 2014, 7 (03): : 774 - 784
- [49] Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model SCIENCE ADVANCES, 2015, 1 (02):
- [50] Spinal muscular atrophy from gene to treatment ABSTRACTS OF PAPERS OF THE AMERICAN CHEMICAL SOCIETY, 2018, 256