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Hereditary transthyretin (ATTR) amyloidosis is a debilitating highly penetrant autosomal dominant disease leading to motor disability within five years and generally fatal within a decade without treatment. Peripheral nerve damage can be isolated, in the absence of cardiac and autonomic involvement. Such a presentation makes it often difficult to distinguish ATTR amyloidosis-related peripheral neuropathy from other acquired peripheral neuropathies of adulthood.
Tegsedi (inotersen) is an antisense oligonucleotide inhibitor of the transthyretin (TTR) protein for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Inotersen is an active ingridients in Tegsedi, inactive ingredients are: purified water (water for injection), hydrochloric acid and or sodium hydroxide for pH adjustment.
Tegsedi is a medicine used to treat the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The most common side effects include: injection site reactions (such as redness or pain at the injection site), nausea, headache, tiredness, low platelet counts (thrombocytopenia), and fever. Tegsedi may cause serious side effects like stroke, immune system problems, liver problems, allergic reactions and eye problems.