patisiran

11/06/2023 - "Patisiran is beneficial for the treatment of TTR cardiac amyloidosis."
01/01/2020 - "Altogether, the data across phase I-III clinical trials points to patisiran as an effective and safe drug for the treatment of hATTR amyloidosis. "
10/26/2023 - "In this trial, administration of patisiran over a period of 12 months resulted in preserved functional capacity in patients with ATTR cardiac amyloidosis. "
10/01/2023 - "As a post hoc analysis, the PK and PD results from the current study were compared with prior patisiran studies in healthy volunteers from a Phase 1 study and in patients with hATTR amyloidosis without LT from Phase 2 and 3 studies. "
12/27/2022 - "Patisiran Enhances Muscle Mass after Nine Months of Treatment in ATTRv Amyloidosis: A Study with Bioelectrical Impedance Analysis and Handgrip Strength."

12/01/2019 - "An open-label-extension study is still underway but, based on the positive results, the regulatory agencies granted approval for the treatment of ATTRv with polyneuropathy in Stage I and II.Expert opinion: The patisiran program has demonstrated that substantial TTR concentration reduction is associated with significant and sustained improvement in polyneuropathy scores, quality-of-life profile and several outcome measures that capture the systemic burden of the disease. "
03/01/2019 - "Patisiran significantly improved polyneuropathy and quality of life (QoL) in the phase III APOLLO trial. "
03/01/2023 - "Patisiran treatment improved neurologic function and QOL across a wide range of baseline polyneuropathy severities versus placebo. "
03/01/2020 - "Patisiran halted or reversed polyneuropathy and improved quality of life from baseline in the majority of patients. "
03/01/2023 - "Impact of baseline polyneuropathy severity on patisiran treatment outcomes in the APOLLO trial."

01/01/2018 - "In this trial, patisiran improved multiple clinical manifestations of hereditary transthyretin amyloidosis. "
01/01/2019 - "Association of Patisiran, an RNA Interference Therapeutic, With Regional Left Ventricular Myocardial Strain in Hereditary Transthyretin Amyloidosis: The APOLLO Study."
12/01/2022 - "Indeed, patisiran has been released in 2018 for hereditary transthyretin amyloidosis. "
01/01/2022 - "Italian Real-Life Experience of Patients with Hereditary Transthyretin Amyloidosis Treated with Patisiran."
12/01/2019 - "An evaluation of patisiran: a viable treatment option for transthyretin-related hereditary amyloidosis."

01/01/2022 - "Patisiran elicited a rapid, sustained TTR reduction (median reduction [Months 6 and 12 average], 91.0%; 95% CI: 86.1%-92.3%); improved neuropathy, quality of life, and autonomic symptoms from baseline to Month 12 (mean change [SEM], Neuropathy Impairment Score, -3.7 [2.7]; Norfolk Quality of Life-Diabetic Neuropathy questionnaire, -6.5 [4.9]; least-squares mean [SEM], Composite Autonomic Symptom Score-31, -5.0 [2.6]); and stabilized disability (Rasch-built Overall Disability Scale) and nutritional status (modified body mass index). "
09/01/2020 - ": At 18 months, compared with placebo, patisiran improved Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) score; (least squares [LS] mean difference: -21.1; p = 1.10 × 10-10; improved across all domains), EuroQoL 5-dimensions 5-levels (LS mean difference: 0.2; p = 1.4 × 10-12), EuroQoL-visual analog scale (LS mean difference: 9.5; p=.0004), Rasch-built Overall Disability Scale (LS mean difference: 9.0; p = 4.07 × 10-16) and Composite Autonomic Symptom Score-31(COMPASS-31; LS mean difference: -7.5; p=.0008). "
01/01/2020 - "The Norfolk Quality of Life-Diabetic Neuropathy score and gait speed improved in 51% of the patisiran-treated group in 18 months. "
09/11/2017 - "Secondary objectives are to evaluate the effect of patisiran on Norfolk-Diabetic Neuropathy quality of life questionnaire score, nutritional status (as evaluated by modified body mass index), motor function (as measured by NIS-weakness and timed 10-m walk test), and autonomic symptoms (as measured by the Composite Autonomic Symptom Score-31 questionnaire). "
10/01/2020 - "In APOLLO (n = 225), patisiran-treated groups showed stabilization or improvements in neurological function (modified Neuropathy Impairment Score +7) and quality of life (Norfolk Quality of Life-Diabetic Neuropathy questionnaire) at 18 months, regardless of prior transthyretin stabilizers. "

06/01/2022 - "The two cases showed that treatment with patisiran for the patients with advanced stage of His88Arg ATTRv cardiomyopathy was unable to stop the progression of HF. "
02/01/2023 - "Patisiran should be considered in ATTRh cardiomyopathy with polyneuropathy. "
10/01/2022 - "Preliminary data show that therapy with patisiran improves the cardiac phenotype rather than only inducing disease stabilization in patients with ATTR variant polyneuropathy and concomitant ATTR cardiomyopathy, and this drug is being evaluated in a phase III clinical trial in patients with ATTR cardiomyopathy. "
01/01/2023 - "Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy."
01/01/2021 - "The purpose of this study was to determine the effect of patisiran on the cardiac amyloid load as measured by cardiac magnetic resonance and extracellular volume (ECV) mapping in cases of transthyretin cardiomyopathy (ATTR-CM). "