UX007 for the treatment of long chain-fatty acid oxidation disorders: Safety and efficacy in children and adults following 24 weeks of treatment

J. Vockley, B. Burton, G. T. Berry, N. Longo, J. Phillips, A. Sanchez-Valle, P. Tanpaiboon, S. Grunewald, E. Murphy, R. Humphrey, J. Mayhew, A. Bowden, L. Zhang, J. Cataldo, D. L. Marsden, E. Kakkis

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