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New Insights into Lysosomal Storage Diseases Pathophysiology is Changing Treatment

Lysosomal & Rare Disorders Research & Treatment Center

New Insights into Lysosomal Storage Diseases Pathophysiology is Changing Treatment

August 10, 2021 – August 9, 2022

NO LONGER AVAILABLE FOR CREDIT

Estimated time to complete: 1.0 hour

Continuing Education Information

This continuing education activity is provided through collaboration between Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for Physicians, Physician Assistants, Nurse Practitioners, Nurses, Genetic Counselors as well as a statement of participation for other participants.

Activity Description

As we gain a better understanding of the pathophysiology of various lysosomal storage diseases, such as Gaucher disease, LAL deficiency disease, Fabry disease, Sanfilippo syndrome, Sly syndrome, etc, we are also unveiling new opportunities to treat these conditions on a more personal level. This 1.0 hour program with Drs. Ozlem Goker-Alpan and Gregory Grabowski reviews the latest research that is reshaping how we will manage individuals with various lysosomal diseases.

Faculty

Ozlem Goker-Alpan, MD
Founder and President
Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)

Gregory Grabowski, MD
Department of Pediatrics
University of Cincinnati College of Medicine
Division of Human Genetics

Learning Objectives

At the end of this activity, participants should be able to:

  • Describe the traditional concepts of the pathophysiology of LSDs
  • Describe the new research into the pathophysiology of LSD at the cellular and system level
  • Describe how new information can improve how patients are managed

Disclosures

AffinityCE, LDRTC, and CheckRare CE staff as well as planning and review committees have no financial interests to disclose and do not recommend the use of products outside of the FDA approved labelling.

Faculty disclosures

Dr. Ozlem Goker-Alpan
BioEvents: Speaker
Chiesi, Sanofi Genzyme, Takeda, Amicus, Prevail, Freeline: Consultant
Chiesi, Takeda, Sanofi Genzyme, Amicus, Avrobio, Idorsia, 4DMT, Sangamo, Protalix: Grant PI
Chiesi, Takeda, Sanofi Genzyme, Amicus, Avrobio, Idorsia, 4DMT, Sangamo, Protalix
Takeda, Prevail Therapeutics, Avrobio: Consultant
Takeda, Sanofi Genzyme, Pfizer, Centogene: Grants

Gregory Grabowski, MD
Asklepios Biotherapeutics: Clinical SAB 2018, SAB 2020-
CCHMC: Owns my LAL and GCase patents
Sanofi Genzyme: Consultant 1989-2013, 2020
Kiniksa Pharmaceuticals, Ltd.– CSO (2015-2019): Founder/Stockholder
Lysosomal Therapeutics, Inc.: SAB 2015-2017, Consultant 2018, 2019
Michael J. Fox Foundation: Chair, GBA1 Structure Workshop 2019, Consultant
Prevail Therapeutics: Consultant 2017-2021
RallyBio, Inc: Consultant, 2020

Conflict of interest for Dr. Goker-Alpan and Dr. Grabowski was resolved through peer review of content by a non-conflicted reviewer.

Physicians

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.

AffinityCE designates this enduring activity for a maximum of 1 hour of AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Physician Assistants

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.

AffinityCE designates this enduring activity for a maximum of 1 hour of AMA PRA Category 1 Credit™. Physician Assistants should claim only the credit commensurate with the extent of their participation in the activity.

Nurses

Continuing Nursing Education is provided for this program through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center’s Commission on Accreditation (ANCC). This activity provides a maximum of 1 hour of continuing nursing education credit.

Nurse Practitioners

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.

AffinityCE designates this enduring activity for a maximum of 1 hour of AMA PRA Category 1 Credit™. Nurse practitioners should claim only the credit commensurate with the extent of their participation in the activity.

Genetic Counselors

Category 2 CEU

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.

AffinityCE designates this enduring activity for a maximum of 1 hour of AMA PRA Category 1 Credit™. This activity meets Category 2 CEUs requirements of the National Society of Genetic Counselors (NCGC).

Other Professionals

All other health care professionals completing this continuing education activity will be issued a Statement of participation indicating the number of hours of continuing education credit. This may be used for professional education CE credit. Please consult your accrediting organization or licensing board for their acceptance of this CE activity.

Commercial Support

Educational Support for this activity was provided by Takeda, Chiesi, Ultragenyx Pharmaceutical, and Spark Therapeutics.

Participation Costs

There is no cost to participate in these CE sessions.

CME Inquiries

For all CME policy-related inquiries, please contact us at ce@affinityced.com.
Send customer support requests to cds_support+ldrtc@affinityced.com.

New Insights into Lysosomal Storage Diseases Pathophysiology is Changing Treatment

Slides