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hereditary transthyretin (hATTR) amyloidosis

The US Food and Drug Administration (FDA) has approved the first RNA-based therapy for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Read more

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RNAi therapeutics company, Alnylam Pharmaceuticals, has revealed that the CHMP has adopted a positive opinion on the use of patisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or 2 polyneuropathy Read more

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Akcea Therapeutics and Ionis Pharmaceuticals have announced that Tegsedi (inotersen) has received European marketing authorisation approval for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hATTR Read more

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Akcea Therapeutics, has announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending the use of its orphan drug for the treatment of Stage 1 or Stage 2 polyneuropathy in adults with hereditary Read more

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