This CME program provides information on best practices to manage children with lysosomal disorders who have been identified by newborn screening. WIth the wide range of symptoms and severities that present for these rare conditions, it is not always certain when the best time to start treatment is in these patients.

Continuing Education Information
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.

To obtain credit, visit https://checkrare.com/learning/p-transforming-clinical-outcomes-with-early-treatment-of-lysosomal-disorders/ 

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 MD
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.

Learning Objectives
At the end of this activity, participants should be 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

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...