Annual Symposium on Lysosomal Storage Disorders


Receive 6.50 AMA PRA Category 1 Credits™

Date: Friday, Dec. 8, 2017

Location: Cedars-Sinai Advanced Health Sciences Pavilion, Los Angeles, California 90048

Course directors: Barry E. Rosenbloom, MD, FACP, Natalie M. Gallant, MD, FACMG

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Why You Should Attend

Genetic deficiencies of lysosomal components, most commonly enzymes, are known as lysosomal storage disorders and lead to lysosomal dysfunction, broadly affecting peripheral organs and the central nervous system, debilitating patients and frequently causing fatality. This continuing medical education conference will update members of the multidisciplinary care team on most recent advances in identification, diagnosis, pathophysiology, management and treatment of a wide range of lysosomal storage disorders.

The theme of the course is translation of scientific breakthroughs to the bedside. Highlights include an update on newborn screening for Gaucher disease, pathophysiology and promise of gene therapy for juvenile Batten disease, and presentation and discussion of various clinical cases. Cardiovascular disease in MPSII and topics related to additional new treatment modalities, including intrathecal enzyme replacement, will also be explored. 

Who Should Attend

This activity is intended for clinicians, researchers and healthcare professionals who wish to explore specific areas of research, diagnosis and the clinical implications for patients with or suspected of having lysosomal storage disorders.