The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment's ability to help boys with the inherited disease. Still, the FDA's outside experts voted narrowly in favor of making the gene therapy available on a preliminary basis, noting the deadly nature of Duchenne and …
During her pregnancies, doctors encouraged Brittany Horsey to take a preterm birth drug called Makena. “In the vast majority of accelerated approvals,” it said, the drug’s clinical benefits were later verified through the confirmatory studies it required. Exondys 51 Duchenne muscular dystrophy $300,000 per year Aduhelm Alzheimer’s disease $28,200 per year Makena Preterm birth $13,000 per pregnancy For decades, the …
WASHINGTON — When a controversial Alzheimer’s drug won U.S. approval, surprise over the decision quickly turned to shock at how long it might take to find out if it really works — nine years. “We think nine years is unacceptable and our expectation is that it will happen in a much shorter time frame,” said Maria Carrillo of the Alzheimer’s …
