The American Journal of Managed Care2 小时
Key Considerations for Choosing the Right SMA Treatment
FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration ...
生物通1 个月
Nusinersen 治疗小儿脊髓性肌萎缩症：疗效、安全性与经济学评价新突破
为探究 Nusinersen 治疗小儿脊髓性肌萎缩症（SMA）的疗效、安全性及经济影响，南京医科大学附属儿童医院研究人员开展回顾性研究，发现该药疗效显著、安全性良好，纳入医保后经济可行性提高，对临床治疗有重要指导意义。 脊髓性肌萎缩症（Spinal Muscular Atrophy ...
Nature3 年
The expanding frontier of nucleic acid therapeutics
For example, Biogen’s nusinersen, an ASO therapy for the fatal neuromuscular condition spinal muscular atrophy (SMA), can reduce by half the likelihood of death or the need for mechanical ...
来自MSN2 个月
Ionis stock rises on FDA, EMA nod for SMA drug dose increase
Biogen Inc. (NASDAQ:BIIB), which licensed the rights to nusinersen from Ionis Pharmaceuticals, stated that the new regimen could offer meaningful benefits to patients. The higher dose comprises ...
Pharmabiz14 天
Novartis announces positive data from phase III study of intrathecal onasemnogene ...
Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA: Basel Friday, March 21, 2025, 11:00 Hrs [IST] N ...
Pharm Exec14 天
Phase III Trial Shows Novartis’ Gene Therapy Significantly Improves Spinal Muscular ...
The trials show that patients administered OAV101 IT achieved a statistically significant improvement in Hammersmith Functional Motor Scale Expanded (HFMSE) scores compared to a sham control group, as ...