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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.

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Unfolding The Folds Of Transthyretin

Delveinsight

The present hATTR treatment market constitutes compounds that stabilize the TTR tetramers, a TTR-directed small interfering RNA, and a TTR-directed antisense oligonucleotide, along with supportive therapies. Gene-silencing Therapies. hATTR market : In The Pipeline.