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NICE recommends Akcea's Tegsedi for rare, life-threatening progressive disease

05:30 EDT 17 Apr 2019 | Pharmafile

Akcea Therapeutics has shared the news that Tegsedi (inotersen), its antisense oligonucleotide (ASO) inhibitor of human transthyretin (TTR) production, has been recommended by NICE for use on the NHS in the treatment of stage 1 or stage 2 polyneuropathy with hereditary transthyretin amyloidosis (hATTR).

hATTR is a rare condition caused by abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in the body’s tissues and organs. It progresses rapidly and can be fatal, with a median survival of just 4.7 years from diagnosis.    

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Original Article: NICE recommends Akcea's Tegsedi for rare, life-threatening progressive disease

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