On February 10 – 13, 2020, leading lysosomal disease researchers will come together to share the latest scientific information about rare lysosomal conditions at WORLDSymposium. The annual event provides an opportunity for basic, transitional, and clinical researchers to interact with each other as well as with patients to help advance therapies for these rare diseases.

WORLDSymposium began in 2002 as the brainchild of Chester B. Whitley, PhD, MD, Professor, Department of Pediatrics, at the University of Minnesota. 2020’s conference is expected to have over 1,900 participants from 50+ countries in attendance. It is a three-day conference that includes one day focused on basic research, one day on transitional research, and one day on clinical research. There will also be a large array of posters on display throughout the meeting being presented by leading researchers and biotech companies in the lysosomal disease space.

Lysosomal diseases are a group of about 50 rare diseases caused by defects in lysosomal proteins or lysosomal related-proteins, which results in dysfunction of lysosomes. Most of these diseases are caused by the genetic absence of a single catabolic enzyme, causing accumulation of the enzyme’s substrate within the lysosome.

Some of the more common lysosomal storage disorders include:

  • Gaucher disease
  • Fabry disease
  • Niemann-Pick disease
  • Pompe disease
  • Tay-Sachs disease
  • Mucopolysaccharide Storage (MPS) Diseases (Hurler syndrome [MPS I], Hunter syndrome [MPS II], Sanfilippo syndrome [MPS III], Morquio A syndrome [MPS IV], Maroteaux-Lamy syndrome [MPS V]), Sly syndrome [MPS VII])

CheckRare will be on location at the conference to interview some of the biggest names in the rare disease space, including Drs Dwight Koebel, Priya Kishnani, David Kronn, and more. If you are planning to be in attendance, be sure to reach out to us and say hi.

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