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

pharmaphorum

2020 also saw some of the first “tumour agnostic” cancer drugs get to market, with Bayer’s Vitravki (larotrectinib) getting funding in the UK for tumours with confirmed neurotrophic tyrosine receptor kinase (NTRK) gene fusions.”. The antibodies produced as a result lodge in the kidneys, causing severe complications.

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Unfolding The Folds Of Transthyretin

Delveinsight

Hereditary ATTR amyloidosis , also known as Familial ATTR, is an autosomal dominant disease and a result of mutations in the TTR gene, thus it is passed down from one generation to the next one. The hATTR diagnosis is mainly dependent on symptoms and can be confirmed via performing tissue biopsies, genetic testing, and imaging studies.

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NICE backs NHS use of Novartis’ cholesterol drug Leqvio

pharmaphorum

. “The obvious disappointment is that they don’t seem to have thought it cost effective for familial hypercholesterolaemia (FH), where patients have genetically elevated LDL-C and limitations on treatments available. This needs further thought,” he added.

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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs.

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