The Food and Drug Administration on Friday approved a landmark rare disease treatment — the first to rely on a Nobel-prize-winning technique known as RNA interference, which silences disease-causing genes.
The approval is a major accomplishment for Cambridge, Mass.-based Alnylam, which will be marketing the drug, patisiran, as Onpattro and which has been working to bring an RNAi-based therapy to market for more than a decade.
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There are volumes of cases of Friedreich’s ataxia in Louisiana. How do we get the word out to neurologist here?