Accreditation/Credit Designation

Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of 3.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Physicians’ Education Resource®, LLC is approved by the California Board of Registered Nursing, Provider #16669, for 3.0 Contact Hours.

Acknowledgment of Commercial Support

This activity is supported by educational grants from Sanofi Genzyme and Ultragenyx Pharmaceutical Inc.

“D” is for Diagnosis: Solving the Mystery of Lysosomal Storage Disorders

Release Date: July 24, 2020
Expiration Date: July 24, 2021

Activity Overview

This online, on-demand virtual symposium brings together renowned experts in lysosomal storage disorders, inherited metabolic disorders characterized by enzyme deficiencies that affect the function of lysosomes. In this educational program these experts use a case-based format to discuss the latest data regarding management of these disorders, share their perspectives and best practices, and forecast applications of key clinical challenges and questions regarding identification of rare LSDs, including Fabry disease, Pompe disease, mucopolysaccharide storage disease type 1, Niemann-Pick disease (types A and B), and Gaucher disease.

This educational activity is an archive of the live virtual symposium held on March 28, 2020.

Acknowledgement of Support

This activity is supported by educational grants from Sanofi Genzyme and Ultragenyx Pharmaceutical Inc.

Instructions for This Activity and Receiving Credit

  1. Complete the activity (including pre- and post-activity assessments).
  2. Answer the evaluation questions.
  3. Request credit using the drop-down menu.

You may immediately download your certificate.

Target Audience

This activity is intended for non-physician healthcare providers who provide a significant portion of patient care in rare diseases, including genetic counselors, nurses, physician assistants, and nurse practitioners.

Learning Objectives

Upon successful completion of this educational activity, you should be better prepared to:

  • Define the disease manifestations and clinical implications of LSDs
  • Distinguish key differentiating signs and symptoms of each LSD to ensure accurate and timely diagnosis
  • Apply recent clinical trial data in the context of individual patient care for improved outcomes in patients with LSDs
  • Recognize patient- and therapy-specific factors that may impact medication adherence
  • Integrate knowledge of case-based challenges into clinical practice for the diagnosis and prognosis of LSDs

Faculty, Staff, and Planners’ Disclosures

In accordance with ACCME Guidelines, PER® has identified and resolved all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.


Dawn Jacob Laney, MS, CGC, CCRC
Dawn Jacob Laney, MS, CGC, CCRC
Assistant Professor/Genetic Counselor
Director, Emory Genetic Clinical Trials Center
Program Leader, Emory Lysosomal Storage Disease Center
Emory University
Atlanta, GA

Disclosures: Grant Research Support: Grant funding from Sanofi Genzyme for ISS; Research Coordinator for industry sponsored studies from Amicus Therapeutics, Sanofi Genzyme, Protalix BioTherapeutics, Sangamo Therapeutics, Takeda/Shire; Consultant: Takeda/Shire, Protalix BioTherapeutics, Amicus Therapeutics, Sanofi Genzyme; Other: Serve on the Fabry Registry NA Board of Advisors; Co-Founder, ThinkGenetic, Inc.

Kyrieckos (Kirk) Aleck, MD
Kyrieckos (Kirk) Aleck, MD
Professor of Child Health
University of Arizona College of Medicine Arizona Division of Genetics and Metabolism Phoenix Childrens Hospital
Phoenix, AZ

Disclosures: Grant Research Support: Sanofi Genzyme, Takeda; Consultant: Sanofi Genzyme, Takeda, Ultragenyx, Alexion; Speakers Bureau: Protalix BioTherapeutics, Biomarin.

Stephanie Cagle, MS, CGC
Stephanie Cagle, MS, CGC
Certified Genetic Counselor
Department of Laboratory Medicine and Pathology
Mayo Clinic
Atlanta, GA

Disclosures: Stephanie Cagle, MS, CGC, has no relevant financial relationships with commercial interests to disclose.

The staff Physicians' Education Resource®, LLC (PER®) have no relevant financial relationships with commercial interests to disclose.

Off-Label Disclosure and Disclaimer

This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a healthcare professional relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER® or any company that provided commercial support for this activity.

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