Malattie rare Approfondimento Quali sono le cause della cTTP? 2 min ● 22.12.2025 La porpora trombotica trombocitopenica congenita (cTTP) è una rara malattia autosomica recessiva causata da mutazioni del gene ADAMTS13 che...
Malattie rare Articolo scientifico Clinical Efficacy and Real-World Effectiveness of Fabry Disease Treatments: A Systematic Literature Review 1 min ● 05.12.2025 This systematic literature review aimed to identify studies assessing the clinical efficacy and real-world effectiveness of current and emerging...