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)
Jerry Vockley*, Barbara Burton, Gerard T. Berry, Nicola Longo, John Phillips, Amarilis Sanchez-Valle, Pranoot Tanpaiboon, Stephanie Grunewald, Elaine Murphy, Alexandra Bowden, Wencong Chen, Chao Yin Chen, Jason Cataldo, Deborah Marsden, Emil Kakkis
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