Gaucher disease (GD): Updates from recent research findings

Gaucher disease (GD): Updates from recent research findings is organized by ScientiaCME.

Release Date: April 28, 2019
Expiration Date: April 28, 2021

Accreditation:
Credit Designation: ScientiaCME designates this educational activity for a maximum of 1.25 AMA PRA Category 1 Credit(s)™ toward the AMA Physician's Recognition Award. Physicians should only claim credit commensurate with the extent of their participation in the activity.

Pharmacists:
ScientiaCME is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 1.25 hours (0.125 CEUs) of continuing education. Proof of participation will be posted to your NABP CPE profile within 4 to 6 weeks to participants who have successfully completed the post-test. Participants must participate in the entire presentation and complete the course evaluation to receive continuing pharmacy education credit. ACPE # 0574-0000-19-013-H01-P. CPE credit effective 4/28/19 expires 4/28/21. 

Description:
In this online self-learning activity:
Gaucher disease (GD) is characterized by a deficiency of the lysosomal enzyme glucocerebrosidase, resulting in the accumulation of sphingolipids throughout the body but most manifesting prominently in the bones. GD is subcategorized based on clinical features: type 1 GD is the non-neuronopathic form and affects mainly the inner organs, while types 2 and 3 are the acute and sub-acute neuropathic forms, whose pathology manifests predominantly within central nervous system. GD impacts about 1 in 75,000 births, making it one of the most common lysosomal storage diseases. One of the first of GD’s complications is the chronic anemia and a persistent bleeding risk. Another is the hepatosplenomegaly, which may be a part of the initial clinical presentation, as may the anatomical abnormalities of bone deformities and stunted growth.

Objectives:
By the end of the session the participant will be able to:
• Describe the pathogenesis, clinical presentations, complications, and epidemiology of Gaucher disease including updated recently presented material
• Describe principles and problems regarding screening for and diagnosing Gaucher disease that can applied to patient cases
• Describe emerging therapies for Gaucher disease based on research recently presented
• Design and implement appropriate therapeutic plans for treatment of Gaucher disease based on research recently presented