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Hereditary thyroxine protein amyloid degeneration is a rare, hereditary, reciditive disease caused by mutations in the transmethyl (TTR) gene.
Patisiran is an RNA interference therapy that inhibits TTR production and has the potential to treat amyloid degeneration of hereditary thyroxine protein with multiple neuropathy.
recently assessed the safety and effective of Patisiran's long-term treatment.
OLE (Phase III) trials were conducted in 43 hospitals or clinical centres in 19 countries, with patients who had previously completed the APOLLO (Phase III) or OLE (Phase II) study and were resistant to Patisiran treatment, injecting Patisiran 0.3 mg/kg every 3 weeks and planning to last for 5 years.
assessment of the efficacy of the study included multiple neuropathy scores (improved neuropathic damage scores of 7 (mNIS)), quality of life, autoitic neurological symptoms, nutritional status, disability, walking status, motor function, and heart stress.
as of September 24, 2018, 126 (92%) of the 137 patients from the APOLLO-Patisiran group, 38 (78%) from 49 patients in the APOLLO-placebo group, and all 25 (100%) from the OLEL-Patisiran group completed a 12-month assessment.
patients were treated with Patisiran, the improvement in mNIS-7 was sustained (average change in the APOLLO-Patisiran group -4.0; average change in the OLE-patisiran group in phase II was -4.7).
total, 204 (97 per cent) of the 211 patients reported adverse events, 82 (39 per cent) reported serious adverse events and 23 (11 per cent) died.
57 percent in the APOLLO-placebo group, compared with 35 percent in the APOLLO-Patisiran group and 24 percent in the OLEL-Patisiran group.
treatment-related adverse events are mild and moderate infusion-related reactions.
27 percent in the APOLLO-placebo group, compared with 7 percent in the APOLLO-Patisiran group and no deaths in the OLE-Patisiran group II.
in a 12-month long-term study, patients with genetic thyroxine protein amyloid degeneration were treated with Patisiran to maintain clinical improvement and good safety.
