pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. The post FDA approves Alnylam’s ultra-rare disease drug Oxlumo appeared first on.

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pharmaphorum

DECEMBER 23, 2020

There was some steady progress in neurology – in February FDA approved Lundbeck’s eptinezumab prophylactic treatment for migraine, the last from a gang of four drugs from a new class. Biogen and Denali will co-market the lead LRRK2 product in the US and China, and Biogen will commercialise in all other markets.

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The Pharma Data

NOVEMBER 24, 2020

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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Drug Discovery World

AUGUST 1, 2023

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. Though some genetic mutations are harmful, others can be protective.

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