onasemnogene abeparvovec (Zolgensma) Report issue

Gene therapy Orphan Drug FDA Approved FDA Breakthrough Therapy FDA

Active Ingredient History

NOW
  • Now
Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. It works by providing a new copy of the SMN gene that produces the SMN protein.   Wikipedia

More Chemistry
avxs-101

Feedback

Data collection and curation is an ongoing process for CDEK - if you notice any information here to be missing or incorrect, please let us know! When possible, please include a source URL (we verify all data prior to inclusion).

Report issue