Zito Anna, De Pascalis Antonio, Armeni Annarita, Ria Paolo, Barbarini Leonardo, Caggiula Marcella, My Filomena, Barbarini Silvia, Trianni Giorgio, Napoli Marcello
Unità Operativa Nefrologia e Dialisi, Ospedale Vito Fazzi, Lecce.
Unità Operativa Neurologia, Ospedale Vito Fazzi, Lecce.
G Ital Nefrol. 2018 Sep;35(5).
Fabry disease (also known as Anderson-Fabry disease, angiocheratoma corporis diffusum, diffuse angiocheratoma) is a rare tesaurismosis linked to the deficiency of the lysosomal enzyme alpha-galactosidase A, required for the physiological catabolism of glycosphingolipids. The related clinical signs show a multisystemic feature and define a degenerative and disabling pathology, whose approach requires a close multidisciplinary specialist collaboration. Currently, the renewed interest in the disease is aimed at the need to provide an early diagnosis, in order to early begin the enzyme replacement therapy and to slow down or avoid the establishment of irreparable organ damage. For this reason, the diagnostic suspicion becomes crucial and arises from the careful observation and research of the symptoms, together with the anamnesis and the overall clinical evaluation of the patient.
法布里病(也称为安德森 - 法布里病、弥漫性躯体血管角质瘤、弥漫性血管角质瘤)是一种罕见的贮积症,与溶酶体酶α - 半乳糖苷酶A缺乏有关,该酶是糖鞘脂生理分解代谢所必需的。相关临床症状具有多系统特征,定义了一种退行性和致残性病理状况,其治疗需要多学科专家密切协作。目前,对该疾病重新产生的兴趣旨在满足早期诊断的需求,以便尽早开始酶替代疗法,并减缓或避免不可修复的器官损伤的发生。因此,诊断怀疑至关重要,它源于对症状的仔细观察和研究,以及患者的病史和全面临床评估。