FDA Committee Unanimous on Two Gene Therapies
An FDA advisory committee gave a unanimous thumbs up to two new gene therapies, one for beta-thalassemia and the other for a rare but fatal neurologic disorder.
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An FDA advisory committee gave a unanimous thumbs up to two new gene therapies, one for beta-thalassemia and the other for a rare but fatal neurologic disorder.
New drugs such as emicizumab (Hemlibra) are transforming the lives of patients with hemophilia A, and more are on their way. "It's an amazing time to be a hemophilia provider," says Alice D. Ma, MD.
/PRNewswire/ -- The "Genetic Testing. Global Market Forecasts for Applications and Technologies. Updated for COVID-19 Pandemic impact With Executive and...
First responders who were exposed to toxic dusts and fibers in the aftermath of the World Trade Center terror attacks show increased levels of clonal hematopoiesis linked to the development of blood cancers.
Around 5000 patients are likely to benefit from the drug over the next 3 years.