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Krabbe disease

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Ongoing discussions - Krabbe disease

Living with diseases of nutrition, metabolism and endocrine glands

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- US Approves First RNA Interference Drug
Alnylam’s Onpattro (patisiran) can treat a rare hereditary disease by silencing a specific gene. The first therapy based on RNA interference (RNAi) gene silencing has been approved by the US Food a

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US Approves First RNA Interference Drug