C&EN s Year in Pharma 2023 acs.org - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from acs.org Daily Mail and Mail on Sunday newspapers.
For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.
Over a 52-week treatment period, the gene therapy demonstrated robust evidence for a clinically meaningful benefit, including across several prespecified functional secondary end points vs placebo.