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Unfolding The Folds Of Transthyretin

Delveinsight

Gene-silencing Therapies. Silencing or modifying gene TTR aims to reduce the effect of both variant and wild-type TTR gene, thus reducing hepatic production by targeting its mRNA. The past years have witnessed a shift from off-label and symptomatic therapies to approved TTR stabilizers and gene-silencing therapies.

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2020 review – Pharma’s progress outside of COVID-19

pharmaphorum

Pharma companies have also made headway in rare diseases as several pipeline projects came to fruition. In summer, Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder Oxlumo was approved in the European Union and the US.