1. FDA Approves Denali's Hunter Syndrome Drug Amid Stricter Scrutiny on Rare Disease Therapies
The FDA has approved Denali Therapeutics' new drug for Hunter syndrome, a significant decision that arrives as the agency tightens its standards for rare disease treatments. This approval for the drug, named Avlayah, stands in stark contrast to the agency's recent rejection of a competing gene therapy from Regenxbio, w...