WORLDSymposium Toolkit 2015

Patient Reported Outcomes and Measures of Disease Severity in Lysosomal Disease: This program provided a discussion of challenges to data collection and ways to overcome these challenges to produce valid and reliable research data. There were short presentations in the context of a panel discussion, along with time for audience Q&A.

WORLDSymposium 2015 Program

Following the opening remarks, presentations in the morning and afternoon sessions will discuss innovations in technology and how they can be applied to early diagnosis for lysosomal conditions, progress in gene therapy, and exploitation of differences at the cellular level that may indicate early disease state. On Tuesday, the noon hour is reserved for a meeting with the WORLDSymposia Council of Patient Advocates (COPA)™ that focuses on patient involvement in research planning and subject recruitment. View full program.

WORLDSymposium 2015 Keynote Speaker

Laurie Muldowney, MD, (US FDA), Delivered Keynote at WORLDSymposium 2015 on Wednesday, February 11, 2015. Dr. Muldowney is Medical Officer for the Division of Gastroenterology and Inborn Error Products at CDER/FDA. Read speaker bio.