Giuseppe Vita

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

David Adams, Alejandra González‐Duarte, William O’Riordan et al. · New England Journal of Medicine · 2018 · 2.8K citations · Full text

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

Merrill D. Benson, Márcia Waddington‐Cruz, John L. Berk et al. · New England Journal of Medicine · 2018 · 1.4K citations · Full text

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Craig M. McDonald, Craig Campbell, Ricardo Erazo Torricelli et al. · The Lancet · 2017 · 446 citations · Full text

Rare Diseases, Urology, Clinical Trials +5

Ascorbic acid in Charcot–Marie–Tooth disease type 1A (CMT-TRIAAL and CMT-TRAUK): a double-blind randomised trial

Davide Pareyson, Mary M. Reilly, Angelo Schenone et al. · The Lancet Neurology · 2011 · 260 citations · Full text

Medicine, Ascorbic Acid, Dental Disease +1

miR-21 and 221 upregulation and miR-181b downregulation in human grade II–IV astrocytic tumors

Alfredo Conti, M. Aguennouz, Domenico La Torre et al. · Journal of Neuro-Oncology · 2009 · 229 citations

Neuro-oncology, Glioma, Medicine +12