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US FDA Approves Ionis-AstraZeneca To Treat Rare Nerve Disease

The US FDA has approved Ionis-AstraZeneca to treat rare nerve diseases. The US Food and Drug Administration approved Ionis Pharmaceuticals and partner AstraZeneca's drug to treat rare nerve diseases on Thursday. ....

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Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy

This open-label, single-group, phase 3 trial evaluates eplontersen, an investigational ligand-conjugated antisense oligonucleotide, in adults with hereditary am ....

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9 Highly Anticipated FDA Decisions in Second Half of 2023

After a series of milestone approvals in the first half of 2023, the FDA is slated to decide on four more firsts before the year’s end. ....

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Aiming to catch Alnylam, AstraZeneca & Ionis plan FDA filing for rare disease drug

The planned FDA submission follows the report from AstraZeneca and Ionis Pharmaceuticals that their partnered drug, eplontersen, met the main goals of a pivotal study in treating nerve pain caused by hereditary transthyretin-mediated amyloidosis. The data come one week after rival Alnylam Pharmaceuticals won FDA approval for its second drug for this rare disease. ....

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