Chao‐Yin Chen

A Randomized, Double‐Blind, Placebo‐Controlled, Phase 3 Trial Evaluating the Efficacy of Burosumab, an Anti‐FGF23 Antibody, in Adults With X‐Linked Hypophosphatemia: Week 24 Primary Analysis

J. S. San Martin, R. Lachmann, H. Cheong, 2018, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial

F. Glorieux, H. Cheong, E. Sochett, 2019, The Lancet.

Burosumab Therapy in Children with X‐Linked Hypophosphatemia

J. S. San Martin, M. Whyte, A. Linglart, 2018, The New England journal of medicine.

Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial

F. Glorieux, H. Cheong, E. Sochett, 2019, The Lancet.

Growth Curves for Children with X-linked Hypophosphatemia

J. S. San Martin, A. Rogol, M. Whyte, 2019, The Journal of clinical endocrinology and metabolism.

Rickets severity predicts clinical outcomes in children with X-linked hypophosphatemia: Utility of the radiographic Rickets Severity Score.

J. S. San Martin, J. Pettifor, A. Creo, 2019, Bone.

Burosumab versus continuation of conventional therapy in children with X-linked hypophosphatemia: a randomised, active-controlled, open-label, phase 3 trial

Farzana, F. Glorieux, H. Cheong, 2020 .

Individual heat map assessments demonstrate vestronidase alfa treatment response in a highly heterogeneous mucopolysaccharidosis VII study population

C. Whitley, P. Harmatz, Raymond Y. Wang, 2019, JIMD reports.

Qualitative Research to Explore the Patient Experience of X-Linked Hypophosphatemia and Evaluate the Suitability of the BPI-SF and WOMAC® as Clinical Trial End Points.

R. Arbuckle, N. Bonner, C. Theodore-Oklota, 2018, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

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)

J. Vockley, B. Burton, N. Longo, 2018, Journal of Inherited Metabolic Disease.

Dietary management and major clinical events in patients with longchain fatty acid oxidation disorders enrolled in a phase 2 triheptanoin study

J. Vockley, N. Longo, Yunming Mu, 2020, Clinical nutrition ESPEN.

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)

J. Vockley, B. Burton, N. Longo, 2018, Journal of Inherited Metabolic Disease.