What’s harder, making new drugs or improving care delivery at scale? An opinionated take on NEJM highlights for July 2018
Marc Herant
July 30, 2018
RNA drugs coming of age Hereditary Transthyretin Amyloidosis (ATTR) is a genetic disease in which one of the alleles of Transthyretin (TTR), a protein produced by the liver and with a role in thyroid hormone metabolism, is mutated resulting in amyloid fiber deposits mainly in nerve and cardiac tissues. Patisiran (Alnylam) and inotersen (Ionis) are both oligonucleotides designed to knock down translation of TTR mRNA, the first through the silencing mechanism, the second through an antisense effect. Phase 3 placebo-controlled studies published back-to-back in the journal show clear efficacy for