Program on Regulation, Therapeutics, and Law, Brigham and Women's Hospital and Harvard Medical School, Boston, MA 02120, USA.
IP-Dhyaan LLP, Dwarka, New Delhi 110075, India.
Sci Transl Med. 2020 Nov 11;12(569). doi: 10.1126/scitranslmed.aay9648.
Evaluating the benefits, risks, and costs of two drugs to treat spinal muscular atrophy raises questions about the future of rare disease medicines.
评估两种治疗脊髓性肌萎缩症药物的获益、风险和成本，引发了对罕见病药物未来的思考。
