9:00 AM EDT: Enzyme Replacement Therapy

Lysosomal storage disorders (LSDs) are genetic diseases that result in either the deficiency or malfunction of particular enzymes responsible for the metabolism of waste material at the cellular level. When waste molecules accumulate or are stored in cell lysosomes, they can cause a disruption in cell function, resulting in a variety of symptoms. All LSDs are progressive, meaning that without treatment, they worsen over time. Enzyme replacement therapy is currently available to address the underlying cause of some LSDs. This session will provide an overview of LSDs and discuss treatment options for 2 LSDs.

Learning Outcomes: After the session, attendees will be able to will name 2 LSDs and the enzyme replacement therapy used to treat those disorders.

Contact Hours: 1
CRNI® RUs: 2


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