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Credit Designation

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

Physicians' Education Resource®, LLC designates this live activity for a maximum of 3.0 AMA PRA Category 1 Credit™. 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

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

Saturday, December 14, 2019

Millennium Hilton New York One UN Plaza
One United Nations Plaza
New York, NY 10017
(212) 758-1234


Lysosomal storage disorders (LSDs) are inherited metabolic disorders characterized by enzyme deficiencies that affect the function of lysosomes. LSDs have diverse clinical manifestations with multiorgan involvement and insidious onset. Symptoms are nonspecific and progressive, based on the affected organs. Though LSDs are rare, they have catastrophic and life-altering effects for patients; to improve patient outcomes in these patient populations, it is imperative that clinicians integrate the newest research and guidelines into practice to ensure evidence-based selection of therapy and appropriate management of care. This educational program will discuss the latest data, share perspectives and best practices, and forecast applications of key clinical challenges and questions regarding identification of rare LSDs.

Acknowledgement of Commercial Support

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

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.

Educational Objectives

Upon completion of this activity, learners should be able 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

Program Chair

Dawn Jacob Laney
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


2 Ways to Register
  1. Online: click here.
  2. Phone: Please call (888) 949-0045 or (609) 378-3701.
Physicians' Education Resource®
2 Clarke Drive
Suite 110
Cranbury, NJ 08512
Registration Fees
  Advanced On-Site
Physicians $39 $39
Fellows* $39 $39
Nurses, PAs, Other Health Care Professionals $39 $39
Industry** $79 $79
* FELLOWS registration must be accompanied by a letter from your director/chair stating current fellowship for discount. Cannot be combined with other discounts/coupon codes.
** INDUSTRY is defined by PER® as any person employed by a for-profit organization, including biotech, financial, and pharmaceutical.
For registration assistance, please email, or call (888) 949-0045 or (609) 378-3701.

Physicians' Education Resource®, LLC (PER®) fully complies with the legal requirements of the ADA and the rules and regulations thereof. If any participant in this educational activity is in need of accommodations, please notify us in order to receive service. Please call (609) 378-3701.

Physicians' Education Resource®, LLC reserves the exclusive rights to all recordings or reproductions of the conference and supporting materials. Unauthorized recording, by any means, is expressly prohibited.  This includes, but is not limited to, recording of presentations or reproductions of supporting audio/visual materials, exhibits, and other supporting Continuing Education materials.

By attending a PER® conference, meeting or related activity ("Events"), all participants, attendees, exhibitors, sponsors, and guests ("you") create an agreement between you and PER® regarding the use and distribution of your image, including but not limited to your name, voice, and likeness ("Image").  By attending the Events, you acknowledge and agree that photographs, video, and/or audio recordings may be taken of you and you grant permission to PER® (and its agents) to utilize, in perpetuity, your Image in any electronic or print distribution, or by other means hereinafter created, both now and in the future, for any lawful purpose as determined by PER®.

Meeting information is accurate at the time of posting.

PER® complies with the Physician Payments Sunshine Act as part of the Affordable Care Act. Accordingly, we may be required to collect information on transfers of value provided to any covered recipient under the Act.


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