Rare Diseases & the Non-Physician Provider: Expert Guidance on Recognizing & Managing LSDs

Released On
September 3, 2020

Expires On
September 3, 2021

Media Type

Completion Time
60 minutes

Gastroenterology, Genetics, Neonatology, Neurology, Obstetrics & Gynecology, Pediatrics, Primary Care, Pulmonology

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Jointly provided by the Postgraduate Institute for Medicine and Enquiring Minds, LLC.

This activity is supported in part by an independent education grant from Sanofi Genzyme.

Credit Available

  • Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)
  • Nurses — maximum of 1.0 Nursing Contact Hour

All other health care professionals completing this course will be issued a statement of participation.

Target Audience

This activity is intended for genetic counselors, physician assistants, nurse practitioners and registered nurses engaged in the care of patients with rare diseases and lysosomal storage disorders.

Program Overview

Make real-world decisions and implement expert guidance as you answer diagnostic and therapeutic questions

  • Differential diagnosis: common characteristics and symptom overlap, spectrum of disease, target patient populations
  • Available and investigational treatments: target patient populations, safety-efficacy profiles, evidence-based recommendations
  • Patient-centered considerations: newborn screening and laboratory testing, potential familial impact, timing and types of treatment, adherence, communication

Learning Objectives

After completing this activity, the participant should be better able to:

  • Describe common characteristics of lysosomal storage disorders and recommend appropriate next steps for determining accurate diagnoses
  • Review and apply the latest clinical evidence, guidance and utility of available and investigational treatments for lysosomal storage disorders, including target patient populations and safety-efficacy profiles
  • Address clinical, individual and familial considerations when applying patient-individualized approaches to diagnosis, treatment and assessment


Dawn A Laney, MS, CGC, CCRC

Assistant Professor and Director
Emory Genetic Clinical Trials Center
Program Leader
Emory Lysosomal Storage Disease Center
Genetic Counselor
Emory University School of Medicine
Atlanta, GA

Kara Anstett, MS, CGC

Genetic Counselor
Comprehensive Neurofibromatosis Center
Lysosomal Storage Disorders Program
Department of Neurology
NYU Langone Health
New York, NY

Joint Accreditation Statement

In support of improving patient care, this activity has been planned and implemented by the Postgraduate Institute for Medicine and Enquiring Minds, LLC. 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.

Credit Designation

Physician Continuing Medical Education
The Postgraduate Institute for Medicine designates this enduring material for a maximum of 1 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Genetic Counselors
If you are a Genetic Counselor, you may claim participation in this activity under AMA credit. Your certificate will show that you have participated in this activity and that the activity was awarded for AMA PRA Category 1 Credits™. You may submit this certificate to your governing body for Genetic Counseling continuing education credit. https://www.nsgc.org/category2ceu


The maximum number of hours awarded for this Continuing Nursing Education is 1.0 contact hours.

Disclosures of Conflicts of Interest

Postgraduate Institute for Medicine (PIM) 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 activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of a commercial interest.

Dawn Laney

  • Receives fees from non-CME/CE services received directly from a commercial intereste or their agents: Sanofi Genzyme and Amicus Therapeutics
  • Ownership interest: ThinkGenetic Inc.

Kara Anstett

  • Consulting fees: Amicus
  • Receives fees from non-CME/CE services received directly from a commercial intereste or their agents: Sanofi Genzyme

Planners and Managers

The PIM planners and managers have nothing to disclose. The Enquiring Minds planners, managers and medical writers have nothing to disclose.

Instructions for Participation and Credit

There are no fees for participating and receiving CME credit for this enduring activity. To receive CME credit participants must:

  1. Read the CME/CE information and faculty disclosures.
  2. Participate in the online activity.
  3. Submit the evaluation form.
Certificates will be emailed to the participant

Course Viewing Requirements

Supported Browsers:
Internet Explorer 8.0+ for Windows 2003, Vista, XP, Windows 7, Windows 8.1 and above
Google Chrome 28.0+ for Windows, Mac OS, or Linux
Mozilla Firefox 23.0+ for Windows, Mac OS, or Linux
Safari 6.0+ for Mac OSX 10.7 and above

Supported Phones & Tablets:
Android 4.0.3 and above
iPhone/iPad with iOS 6.1 or above

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.