Jointly provided by the Postgraduate Institute for Medicine and Enquiring Minds, LLC.
Supported by an independent educational grant from Sanofi Genzyme
3 pediatric and adult patient cases that enable clinicians to evaluate and integrate expert guidance on differential diagnosis and therapeutic optimization.
Clinical characteristics and phenotypes
Benefit-risk profiles of available and investigational agents
Mechanisms of action, target patient populations
This activity is intended for neurology, pediatrics, primary care, genetic counselors, nurses, nurse practitioners, and other healthcare practitioners engaged in the care of patients with lysosomal storage disorders.
After completing this activity, the participant should be better able to:
Differentiate key clinical characteristics of lysosomal storage diseases (LSDs) and consider how patient variability plays a role in their diagnosis and treatment
Discuss the implications of delayed diagnosis and treatment of LSDs
Evaluate and integrate current best practices and evolving therapeutic options for treating LSDs, taking into consideration patient and clinical factors that may influence therapeutic approach
Disclosure of Conflicts of Interest
The Postgraduate Institute for Medicine requires instructors, planners, managers, and other individuals who are in a position to control the content of this activity to disclose any real or apparent conflict of interest (COI) they may have as related to the content of this activity. All identified COI are thoroughly vetted and resolved according to PIM policy. PIM is committed to providing its learners with high quality CME/CE activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of a commercial interest.
Heather Lau, MD, MS Director, Lysosomal Storage Disorders Program Associate Director, Division of Neurogenetics Department of Neurology NYU School of Medicine New York, NY
Dr. Lau receives advisory board fees from Amicus Therapeutics, Adult Polyglucosan Body Research Foundation, Avrobio, Biomarin, Chiesi, National Tay-Sachs & Allied Diseases Association, Sanofi Genzyme, Shire; Consulting fees from Actelion, Amicus Therapeutics, ASPA Therapeutics, BioMarin, Chiesi, Pfizer, Prevail Therapeutics, Sanofi Genzyme, Shire, Ultragenyx Pharmaceutical, Inc.; Contracted research fees from Amicus Therapeutics, BioMarin, Mallinckrodt, Pfizer, Protalix, Sangamo, Sanofi Genzyme, Shire, Ultragenyx Pharmaceuticals, Inc.
Kate Anstett, MS, CGC Genetic Counselor Department of Neurology NYU Langone Health New York, NY
Ms. Anstett receives consulting fees from Amicus Therapeutics
Accredited Provider Disclosure
Planners and Managers
The PIM planners and managers have nothing to disclose. The Enquiring Minds, LLC planners and managers have nothing to disclose.
AMA PRA Category 1 Credit(s)TM
In support of improving patient care, this activity has been planned and implemented by the Postgraduate Institute for Medicine and Enquiring Minds, LLC. The Postgraduate Institute for Medicine is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.
The Postgraduate Institute for Medicine designates this enduring material for a maximum of 1.00 AMA PRA Category 1 Credit(s)TM . Physicians should claim only the credit commensurate with the extent of their participation in the activity.
There is no fee for this educational activity.
Disclosure of Unlabeled Use
This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications. The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.
Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.
There are no fees for participating and receiving CME/CE credit for this activity. During the period August 30, 2019 through August 30, 2020, participants must read the learning objectives and faculty disclosures and successfully complete the activity evaluation. Your online certificate will be saved on myCME within your Profile/CME History, which you can access at any time.
System Requirements Windows: Internet Explorer 11, Microsoft Edge (latest version), Google Chrome (latest version), Firefox (latest version) Mac: Safari (latest version), Google Chrome (latest version), Firefox (latest version) Mobile: Safari in Apple iOS 10 or later, Google Chrome in Apple iOS 10 or later, Google Chrome in Android 4.4 or later