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

Concepedia

Publication | Open Access

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

DOI Full Paper Access

78

Citations

26

References

2017

Year

Jerry Vockley, Barbara K. Burton, Gerard T. Berry, Nicola Longo, John G. Phillips, Amarilis Sanchez‐Valle, Pranoot Tanpaiboon, Stephanie Grünewald, Elaine Murphy, Reed Humphrey,

Molecular Genetics and Metabolism

Abstract

In patients with severe LC-FAOD, UX007 interim study results demonstrated improved exercise endurance and tolerance, and were associated with positive changes in self-reported HR-QoL.

References

	Year	Citations

Page 1