Patisiran for treating hereditary transthyretin amyloidosis
Patisiran for treating hereditary transthyretin amyloidosis (Highly specialised technologies guidance [HST10])
Evidence-based recommendations on patisiran (Onpattro) for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy.
Hereditary transthyretin (hATTR) amyloidosis is an ultra-rare condition caused by inherited mutations in the transthyretin (TTR) gene. This causes the liver to produce abnormal TTR protein, which accumulates as deposits in body tissues (amyloidosis). These deposits can disrupt the structure and damage the function of affected tissues.
Because hATTR amyloidosis can affect tissues throughout the body, people may have a range of symptoms relating to 1 or more systems. These can include the autonomic nervous system, peripheral nerves, heart, gastrointestinal system, eyes and central nervous system.
Patisiran (Onpattro, Alnylam) is a ribonucleic acid interference agent that suppresses transthyretin (TTR) production by the liver (including abnormal TTR).
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