Encontrados 2 documentos, a visualizar página 1 de 1
ENSAT registry-based randomized clinical trials for adrenocortical carcinoma
Crona, Joakim; Baudin, Eric; Terzolo, Massimo; Chrisoulidou, Alexandra; Angelousi, Anna; Ronchi, Cristina L; Oliveira, Cristina LamasAdrenocortical carcinoma (ACC) is an orphan disease lacking effective systemic treatment options. The low incidence of the disease and high cost of clinical trials are major obstacles in the search for improved treatment strategies. As a novel approach, registry-based clinical trials have been introduced in clinical research, so allowing for significant cost reduction, but without compromising scientific benefi...
European lipodystrophy registry: background and structure
von Schnurbein, Julia; Adams, Claire; Akinci, Baris; Ceccarini, Giovanni; D’Apice, Maria Rosaria; Gambineri, Alessandra; Hennekam, Raoul C. M.Background: Lipodystrophy syndromes comprise a group of extremely rare and heterogeneous diseases characterized by a selective loss of adipose tissue in the absence of nutritional deprivation or catabolic state. Because of the rarity of each lipodystrophy subform, research in this area is difficult and international co-operation mandatory. Therefore, in 2016, the European Consortium of Lipodystrophies (ECLip) d...