Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome

by Christina Lampe MD, Mohit Jain PhD, Andrew Olaye PhD, Bianca Meesen MSc, Celeste Decker MD, Eugen Mengel MD
Published 2015-04-01

Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study

by Nicola Longo, Roberto Zori, Melissa P. Wasserstein, Jerry Vockley, Barbara K. Burton, Celeste Decker, Mingjin Li, Kelly Lau, Joy Jiang, Kevin Larimore, Janet A. Thomas
Published 2018-07-01