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August 11, 2020 Turpis cursus in hac habitasse platea dictumst quisque sagittis purus. Semper feugiat nibh sed pulvinar proin gravida hendrerit lectus. Optio vel quisquam nobis molestiae iste. Quia inventore est libero amet ea explicabo. Assumenda eligendi repudiandae autem omnis pariatur quam omnis...

Effects of triheptanoin (UX007) in patients with long-chain fatty acid oxidation disorders: Results from an open-label, long-term extension study. Journal of inherited metabolic disease, 44(1), 253–263. https://doi.org/10.1002/jimd.12313

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). Journal of inherited metabolic disease, 42(1), 169–177.

A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) with Phenylketonuria Featuring an Open-Label Randomized Two-Arm (Active vs Diet-Only Control) Design/BMN 165-306

A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) with Phenylketonuria Featuring an Open-Label Randomized Two-Arm (Active vs. Diet-Only Control) Design ClinicalTrials.gov ID NCT05270837 Sponsor BioMarin...

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