Antibody Gene Silencing Genetics Pharma Companies 
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.
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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