Tag «Spinal muscular atrophy»

Onasemnogene abeparvovec オナセムノジーンアベパルボベック

It’s only fair to share…   Onasemnogene abeparvovec オナセムノジーンアベパルボベック DNA (synthetic adeno-associated virus 9 vector scAAV9.CB.hSMN human survivor motor neuron protein-specifying) Zolgensma FDA 2019/5/24 APPROVED CAS: 1922968-73-7 AVXS-101 Spinal muscular atrophy treatment Treatment of Spinal Muscular Atrophy (SMA) Type 1 Gene therapy product Onasemnogene abeparvovec, sold under the trade name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA). …