A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin greatly reduced transthyretin levels in patients with ...
A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin amyloidosis with polyneuropathy. Polyneuropathy stages and disability scores were ...
(RTTNews) - RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (ALNY) announced Tuesday that the European Commission has granted marketing authorization for AMVUTTRA (vutrisiran), an RNAi ...
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