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Chapter 4: Central Nervous System Toggle Pages
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Page last updated:
28 October 2019


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4.12 Oligonucleotide inhibitors

First Line
Second Line
Specialist
Hospital Only
Inotersen
  • Solution for injection pre-filled syringes 284mg/1.5ml

Notes

  1. NICE HST9: Inotersen (Tegsedi) is recommended, within its marketing authorisation, as an option for treating stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis, only if the company provides it according to the commercial arrangement (May 2019).

Transthyretin stabilisers

Patisiran sodium
  • Concentrate for solution for infusion vials 10mg/5ml

Notes

  1. NICE HST10: Patisiran (Onpattro) is recommended, within its marketing authorisation, as an option for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy, only if the company provides it according to the commercial arrangement (August 2019).
Tafamidis
  • Capsules 61mg

Notes

  1. NICE TA984: Tafamidis (Vyndaqel) is recommended, within its marketing authorisation, as an option for treating wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adults, only if the company provides it according to the commercial arrangement (June 2024).