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Current and Emerging Therapies for Lysosomal Storage Disease

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Lysosomal & Rare Disorders Research & Treatment Center

Current and Emerging Therapies for Lysosomal Storage Disease

May 1, 2021 – April 30, 2022

Estimated time to complete: 1.0 hours

Continuing Education Information

This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available for other attendees.

Activity Description

In the first or four webinars focused on the latest developments in lysosomal storage diseases, Drs. Ozlem Goker-Alpan and Gregory Grabowski provide an overview of current and future therapies for these rare genetic conditions (e.g., Gaucher disease, Sly syndrome, Fabry disease, Pompe disease, etc).

Faculty

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

Ari Zimran, MD
Founder and Director, Gaucher Unit, Shaare Zedek Medical Center, Jerusalem, Israel

Learning Objectives

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

  • Describe Rare Disorders
  • Summarize Lysosomal Storage Diseases
  • List the therapeutic approaches and challenges in LSDs
  • Cite the current and emerging therapies for LSDs
  • Describe the limitations of the current treatment options and treatments in development
  • Describe the pros and cons of different treatment modalities

Disclosures

AffinityCE staff; LDRTC staff; and planning and review committees have no financial interests to disclose and do not recommend the use of products outside of FDA-approved labelling.

Faculty disclosures

Dr. Ozlem Goker-Alpan
Dr. Goker-Alpan is a consultant for Chiesi, Takeda, Sanofi/Genzyme, Amicus, Prevail and Freeline.
Receives grants from Chiesi, Takeda, Sanofi/Genzyme, Amicus, AVROBIO, Idorsia, 4DMT, Sangamo and Protalix.
Principle investigator for Chiesi, Sanofi/Genzyme, Amicus, AVROBIO, Idorsia, 4DMT, Sangamo and Protalix.

Dr. Ari Zimran
Dr. Zimran is a consultant for Takeda, Prevail Therapeutics, AVROBIO and Insightec.
The Gaucher Unit receives support from Genzyme/Sanofi for participation in the ICGG Gaucher Registry, from Shire for the GOS Registry and from Pfizer for TALIAS.
The clinic also receives research grants from Takeda, Sanofi, Pfizer and Centogene.

Conflict of interest for Dr. Ozlem Goker-Alpan and Dr. Ari Zimran and 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™. Genetic Counselors should claim only the credit commensurate with the extent of their participation in the activity.

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 Pharmaceuticals USA, Cheisi USA, 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.

Current and Emerging Therapies for Lysosomal Storage Disease

Slides