Results from a 78-week, single-arm, open-label phase 2 study to evaluate UX007 in pediatric and adult patients with severe long-chain fatty acid oxidation disorders (LC-FAOD).

Vockley, Jerry; Burton, Barbara; Berry, Gerard T; Longo, Nicola; Phillips, John; Sanchez-Valle, Amarilis; Tanpaiboon, Pranoot; Grunewald, Stephanie; Murphy, Elaine; Bowden, Alexandra; Chen, Wencong; Chen, Chao-Yin; Cataldo, Jason; Marsden, Deborah; Kakkis, Emil.
J Inherit Metab Dis; 42(1): 169-177, 2019 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-30740733