Amvuttra RNAi Therapeutic Wins FDA Approval for Rare Genetic Protein Disorder
XTalks
JUNE 20, 2022
Alnylam’s Onpattro was its first licensed and approved RNAi therapy for ATTR polyneuropathy. Hereditary ATTR has an autosomal dominant pattern of inheritance and is caused by point mutations in the transthyretin ( TTR ) gene that codes for the transthyretin transport protein found in the plasma and cerebrospinal fluid.
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