IMPROVEMENTS IN MAJOR CLINICAL EVENTS IN CHILDREN TREATED WITH TRIHEPTANOIN FOR LONG-CHAIN FATTY ACID OXIDATION DISORDERS (LC-FAOD)

Kathryn Ray(Ultragenyx Pharmaceutical (United States)), Jerry Vockley(Pittsburgh Public Schools), Xiaoxiao Lu(Ultragenyx Pharmaceutical (United States)), Laura Rosa Pisani(Regenxbio (United States))
Molecular Genetics and Metabolism
March 18, 2022
Cited by 0


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