John Crowley, CEO and Chairman at Amicus Therapeutics, talks about the growth of WORLDSymposium over the past 16 years.

WORLDSymposium is the only conference focused solely on lysosomal diseases. Initially starting out with about 75 people in attendance, this year’s conference had over 2000 attendees. The key driver to the conference’s success is the passion that researchers in this field have towards finding treatments, or better treatments, for the 50+ lysosomal diseases that exist.

Lysosomal diseases are a group of genetic diseases caused by defects in lysosomal proteins or lysosomal related-proteins, which results in dysfunction of lysosomes. Most of these conditions are caused by the absence of a single catabolic enzyme, causing accumulation of the enzyme’s substrate within the lysosome. Over time, tissue-specific substrate accumulations result in a spectrum of symptoms and disabilities that vary by disease. Some of the more well-known diseases include Gaucher disease, Fabry disease, Pompe disease, Batten disease, and Tay-Sachs disease.

At this year’s WORLDSymposium, Crowley was honored to receive the 2020 Roscoe O. Brady Award for Innovation and Accomplishment.  Roscoe Brady, MD, was a pioneer in the lysosomal disease space and was instrumental in developing the first enzyme replacement therapy (for Gaucher disease).

To learn more about the various lysosomal diseases, click here.

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