CME Information FACULTY Gregory M. Pastores, MD Associate Professor of Neurology and Pediatrics New York University School of Medicine Director, Neurogenetics Laboratory Department of Neurology New York University New York, New York INTENDED AUDIENCE This educational activity is intended to educate family/general physicians, internists, hematologists, oncologists, gastroenterologists, hepatologists, orthopedists, clinical geneticists, and other clinicians who may screen and diagnose patients with type 1 Gaucher disease. NEEDS ASSESSMENT Gaucher disease is an autosomal recessive metabolic lipid storage disorder that results in devastating medical complications if undiagnosed or diagnosed late. Type 1 Gaucher disease is the most common form, widely heterozygous in clinical presentation, ranging from asymptomatic to severe symptoms, potentially causing disabling and life-threatening complications. A survey of specialists suggested that only 20% considered Gaucher disease in the differential diagnosis for all of its classic symptoms (ie, cytopenia, hepatosplenomegaly, bone pain), suggesting a great need for physician education to increase the likelihood of prompt detection of Gaucher disease and improve its management. EDUCATIONAL OBJECTIVES At the conclusion of this activity, participants should be better able to... Recognize that type 1 Gaucher disease is a progressive, genetic disorder that may cause disabling and life-threatening complications Associate the widely varied clinical presentations of type 1 Gaucher disease with the condition to enable early differential diagnosis and treatment initiation Differentiate between available classes of treatment for type 1 Gaucher disease, and choose appropriate therapy based on individual patient needs ACCREDITATION AND CERTIFICATION This activity has been planned and implemented in accordance with the Essential Areas and policies of the Accreditation Council for Continuing Medical Education through the joint sponsorship of Dannemiller and CogniMed Inc. Dannemiller is accredited by the ACCME to provide continuing medical education for physicians. Dannemiller designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. There is no charge for this activity. Statements of Credit will be provided by e-mail following activity participation and upon completion and electronic submission of the posttest and evaluation to Dannemiller. A link to the evaluation form is provided upon completion of the activity. If you have any questions about your certificate, please e-mail cme@dannemiller.com. CONTENT REVIEW STATEMENT To resolve identified/potential conflicts of interest, the educational content was fully reviewed by a physician member of the Dannemiller Clinical Content Review Committee who have no financial relationships with commercial interests. The resulting certified activity was found to provide educational content that is current, evidence based and commercially balanced. DISCLOSURE STATEMENT It is the policy of Dannemiller to ensure fair balance, independence, objectivity, and scientific rigor in all programming. All faculty participating in sponsored programs are expected to identify and reference off-label product use and disclose any significant relationships with those supporting the activity or any others whose products or services are discussed. The faculty for this activity have disclosed that there will be discussion about the use of products for non–FDA-approved indications. In accordance with the Accreditation Council for Continuing Medical Education standards, parallel documents from other accrediting bodies, and Dannemiller policy, the following disclosures have been made: Dannemiller Staff Michelle Montgomery, Director of Continuing Education, has nothing to disclose. Mark Nadeau, MD, Content Reviewer, has nothing to disclose. CogniMed Inc. Staff Karla Crenshaw-Williams, PhD, Senior Director, Scientific Affairs, and Medical Writer, has nothing to disclose. Roseann MacLachlan, Senior Program Manager, has nothing to disclose. Elizabeth Stanton, Editor, has nothing to disclose. Faculty Gregory M. Pastores, MD, receives honoraria and research support from BioMarin Pharmaceutical Inc., Genzyme Corporation, Protalix Biotherapeutics, and Shire. The ideas and opinions presented in this educational activity are those of the faculty and do not necessarily reflect the views of Dannemiller and/or its agents. As in all educational activities, we encourage the practitioners to use their own judgment in treating and addressing the needs of each individual patient, taking into account that patient's unique clinical situation. Dannemiller disclaims all liability and cannot be held responsible for any problems that may arise from participating in this activity or following treatment recommendations presented. This activity is supported by an independent educational grant provided by Actelion Pharmaceuticals Ltd. This activity is an enduring material and consists of an online case study. Successful completion is achieved by viewing the material, reflecting on its implications in your practice, and completing the assessment component. The estimated time to complete this activity is 1.0 hour. This activity was originally released December 28, 2012, and is eligible for credit through December 28, 2013. This content was reviewed by an impartial content reviewer and by faculty. Faculty have final editorial control over the piece. © 2012 CogniMed Inc. All rights reserved.        GD14002        December 2012