Delveinsight

AUGUST 6, 2020

The hATTR diagnosis is mainly dependent on symptoms and can be confirmed via performing tissue biopsies, genetic testing, and imaging studies. 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.

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pharmaphorum

DECEMBER 23, 2020

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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Marketing Gene Silencing Gene Drugs 95

Drug Discovery World

OCTOBER 4, 2022

The pandemic might have affected the number of clinical trials taking place for cell and gene therapies, with activity decreasing by around 15% compared to 2020, but major therapies such as Novartis’ gene therapy drug for spinal muscular atrophy (SMA) Zolgensma still made it to market. .

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