Transforming Clinical Outcomes with Early Treatment of Lysosomal Disorders
Start date: Apr 3, 2025
End date: Apr 3, 2026
Estimated time to complete: 1.0 hour
Credits: 1.0 AMA PRA Category 1 Credits™
Other Modules in this Series
Skeletal Involvement in Lysosomal Disorders
Jointly Provided by
This activity was supported by educational grants from Takeda, Sanofi, and Chiesi.
This continuing education activity is provided by AffinityCE and the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC). This activity provides continuing education credit for physicians. A statement of participation is available to other attendees.
Newborn Screening has dramatically transformed how we diagnose and treat many rare lysosomal disorders. For many lysosomal diseases diagnosed at birth however, it is not certain when to start therapy. This CME program examines the evidence available to address how to monitor, and possibly treat, children with lysosomal diseases that were diagnosed by newborn screening or soon after birth.
Learning Objective
After participating in the activity, learners should be better able to:
- Cite the importance of early diagnosis and treatment of lysosomal storage disorders
- List the guidelines for the early treatment of LDs and enhanced integration of newborn screening programs
- Identify key research gaps and priorities and strengthen collaboration among researchers and healthcare professionals
- List the educational resources and support programs for families
Faculty and Disclosures
AffinityCE staff, LDRTC staff, planners, and reviewers, have no relevant financial relationships with ineligible companies to disclose. Faculty disclosures, listed below, will also be disclosed at the beginning of the Program.
Ozlem Goker-Alpan MD
Founder and CMO
Lysosomal & Rare Disorders Research & Treatment Centers
Dr. Goker-Alpan is on the Advisory Board/Consultant for Chiesi, Takeda, Sanofi, Prevail/Lilly, Sparks Therapeutics, Uniqure, Exegenesis, Astellas, Freeline, Team Sanfilippo. She receives grants/research support from Chiesi, Sanofi, Takeda, Prevail/Lilly, Spark Therapeutics, Amicus, Freeline, Sangamo, Cyclo, Odorsia, DMT, Homology, Protaliz. She is on the speaker bureau for Sanofi, Takeda, Amicus, Chiesi
David F. Kronn MD
Associate Professor of Pathology and Pediatrics
New York Medical College
Dr. Kronn is on the Advisory Board for Sanofi. He is also on the speaker bureau for Sanofi. He receives research funding from Sanofi.
Uma Ramaswami FRCPCH, MD
Royal Free London Hospitals & Genetics and Genomic Medicine, University College London
Dr. Ramaswami is on the Advisory Board for Amicus, Chiesi, Sanofi and Takeda. She receives research grants from Chiesi and Intabio.
Liz Jalazo MD
Assistant Professor of Pediatrics and Genetics
University of North Carolina at Chapel Hill
Dr. Jalazo is on the Advisory Board for Sanofi and Ionis.
Lindsay Torrice MSN, CPNP-PC
Assistant Professor of Pediatrics
University of North Carolina at Chapel Hill
Ms. Torrice has no financial relationships to disclose.
Mitigation of Relevant Financial Relationships
AffinityCE adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible companies. All relevant financial relationships for faculty were mitigated by the peer review of content by non-conflicted reviewers before the commencement of the activity.
Accreditation and Credit Designation
In support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. 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.
Physicians
American Academy of CME, Inc., designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Other Professionals
All other healthcare 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.
Participation Costs
There is no cost to participate in this activity.
Hardware/Software Requirements
Windows Requirements: • Operating system: Windows XP Service Pack 2 or later • Browser: Internet Explorer 7 or later, Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connection
Macintosh Requirements: • Operating system: Mac OS X v10.3 or later • Browser: Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connection
CME Inquiries
For all CME policy-related inquiries, please contact us at mailto:[email protected]
Send customer support requests to mailto:[email protected]
Copyright
© 2025. This CME-certified activity is held as copyrighted © by Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) and AffinityCE. Through this notice, Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) and AffinityCE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).
