Remove Clinical Trials Remove FDA Approval Remove Gene Remove Gene Silencing
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Vevye: A New Cyclosporine Solution for Dry Eye Disease

XTalks

The clinical trials consistently showed significant therapeutic improvements in ocular surface damage and associated symptoms, while pioneering visual function improvement analysis”, said John D. Additionally, the clinical trials underscored the favorable tolerability profile of Vevye.

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Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

pharmaphorum

Novo Nordisk also gets rights to the remainder of Dublin-based Prothena’s ATTR amyloidosis programme under the terms of the deal, although PRX004 is the only candidate that has reached the clinical trials stage. All told, the deal could be worth up to $1.2

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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.”. Neurology on the march. Rare disease progress.

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FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

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World RNA Day: What impact RNA it had on drug discovery?  

Drug Discovery World

As per a definition provided by AstraZeneca: “Once inside, ASOs bind with high specificity to target mRNA or pre-mRNA, inducing its degradation – effectively silencing it – to prevent its translation into a detrimental protein product. AstraZeneca’s non-alcoholic steatohepatitis (or ‘NASH’) study is a prime example of the value of ASOs 2.

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