Background: Hereditary transthyretin (ATTRv, v for variant) amyloidosis with polyneuropathy is a rare disease caused by mutations in the transthyretin gene. In ATTRv amyloidosis, multisystem extracellular deposits of amyloid cause tissue and organ dysfunction. Patisiran is a small interfering RNA molecule drug that reduces circulating levels of mutant and wild-type TTR proteins. Prior to its regulatory approval, patisiran was available in Italy through a compassionate use programme (CUP). The aim of this study was to analyse the long-term outcomes of patients who entered into the CUP. Methods: This was a multicentre, observational, retrospective study of patients with ATTRv amyloidosis treated with patisiran. The analysis included change from baseline to 12, 24, 36 and 48 months in familial amyloid polyneuropathy (FAP) stage, polyneuropathy disability (PND) class, neuropathy impairment score (NIS), modified body mass index (mBMI), Compound Autonomic Dysfunction Test (CADT), Karnofsky Performance Status (KPS) scale and Norfolk Quality of Life–Diabetic Neuropathy (QoL-DN) questionnaire. Safety data were also analysed. Results: Forty patients from 11 Italian centres were enrolled: 23 in FAP 1 (6 in PND 1 and 17 in PND 2) and 17 in FAP 2 (8 in PND 3a and 9 in PND 3b) stage. In this population, the mean NIS at baseline was 71.4 (± 27.8); mBMI, 917.1 (± 207) kg/m²; KPS, 67.1 (± 14.0); Norfolk QoL-DN, 62.2 (± 25.2); and CADT, 13.2 (± 3.3). Statistical analysis showed few significant differences from baseline denoting disease stability. No new safety signals emerged. Conclusions: Patisiran largely stabilised disease in patients with ATTRv amyloidosis.

Background: Monoclonal antibodies against the calcitonin gene-related peptide (anti-CGRP mAbs) have been a game-changer in migraine treatment over the last decade. However, data regarding the reduction in the frequency of episodes of migraine with aura are limited, as no trials have been specifically designed to evaluate this outcome. Methods: In this narrative review, we summarized clinical data from both randomized controlled trials (RCTs) and real-world studies (RWSs) on the efficacy and effectiveness of anti-CGRP mAbs in patients with migraine with aura and aura symptoms. Results: Overall, anti-CGRP mAbs can reduce migraine frequency and burden regardless of the presence of aura, with efficacy and effectiveness in both patients with and without aura. A few studies suggested a potential influence on reducing aura occurrence. However, several limitations affect the available studies and prevent definitive conclusions regarding the effects of anti-CGRP mAbs on aura. Conclusions: Further studies specifically aimed at assessing the impact of anti-CGRP therapies on the frequency, duration, and characteristics of aura are necessary. Such research could help elucidate the complex relationship between CGRP and aura.