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 attracted over 20 Exhibitors offering a highly interactive forum for discussion of lysosomal diseases with international researchers and health care practitioners. View Exhibitor and Patient Advocate list.
For the third consecutive year, WORLDSymposium began with Emerging Trends in Lysosomal Biology & Lysosomal Diseases: State-of-the-art for Experts on Monday, February 9, 2015. See agenda and faculty.
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.
In 2015, over 300 scientific abstracts were presented beginning on Monday, February 9, 2015, and remained open throughout the rest of the conference. View Poster List.
7 Satellite symposia were held from Monday, February 9, 2015 to Thursday, February 12, 2015. Click here for the full schedule.
WORLDSymposium 2015 Award for Innovation and Accomplishment was presented to Dr. Stephen C. Groft. Dr. Stephen is a public servant for more than four decades and a pioneer at the Office of Rare Diseases Research. More details.
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.
The WORLDSymposium 2015 New Treatment Awards were presented to Genzyme, Green Cross Corp, Raptor Pharmaceutical Corp and Biomarin Pharmaceutical at the WORLDSymposium banquet on Thursday, February 12. More details.