Current and emerging treatment options for patients with alpha-mannosidosis
Prof. Barbara K Burton considers the best supportive care, role of the multidisciplinary team, current treatment options in the US, and emerging treatments for alpha-mannosidosisview bio and disclosures 3/3 Take CE/CME Test
Overview & Learning Objectives
In this activity, a leading expert will share her insights on the natural history and prevalence of alpha-mannosidosis, how to establish diagnosis, and the current and emerging treatment options for patients with this lysosomal storage disorder
This activity is jointly provided by USF Health and touchIME. read more
This activity has been designed to meet the educational needs of validated lysosomal storage disorder specialists including: geneticists, neurologists and paediatricians involved in the management of alpha-mannosidosis.
USF Health adheres to the Standards for Integrity and Independence in Accredited Continuing Education. All individuals in a position to influence content have disclosed to USF Health any financial relationship with an ineligible organization. USF Health has reviewed and mitigated all relevant financial relationships related to the content of the activity. The relevant relationships are listed below. All individuals not listed have no relevant financial relationships.
Prof. Barbara K Burton discloses: Advisory board or panel: Agios, Alexion, Applied Therapeutics, Maze Therapeutics, Orchard Therapeutics. Consultant: Biomarin, Horizon, JCR Pharmaceuticals, Moderna, Passage Bio, Sanofi, Takeda, Ultragenyx. Grants/research support: Biomarin, Denali, Homology Medicines, JCR Pharmaceuticals, Sangamo, Ultragenyx. Speaker’s bureau: Biomarin, Horizon, Takeda.
Larry J. Dishaw Ph.D. has no financial interests/relationships or affiliations in relation to this activity.
Touch Medical Director
Holly Gilbert has no financial interests/relationships or affiliations in relation to this activity.
USF Health Office of Continuing Professional Development and touchIME staff have no financial interests/relationships or affiliations in relation to this activity.
Requirements for Successful Completion
In order to receive credit for this activity, participants must review the content and complete the post-test and evaluation form. Statements of credit are awarded upon successful completion of the post-test and evaluation form.
If you have questions regarding credit please contact email@example.com
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 a joint providership of USF Health and touchIME. USF Health is accredited by the ACCME to provide continuing medical education for physicians.
USF Health designates this enduring material for a maximum of 0.75 AMA PRA Category 1 CreditTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
The European Union of Medical Specialists (UEMS) – European Accreditation Council for Continuing Medical Education (EACCME) has an agreement of mutual recognition of continuing medical education (CME) credit with the American Medical Association (AMA). European physicians interested in converting AMA PRA Category 1 CreditTM into European CME credit (ECMEC) should contact the UEMS (www.uems.eu)
Advanced Practice Providers
Physician Assistants may claim a maximum of 0.75 Category 1 credits for completing this activity. NCCPA accepts AMA PRA Category 1 CreditTM from organizations accredited by ACCME or a recognized state medical society.
The AANPCP accepts certificates of participation for educational activities approved for AMA PRA Category 1 CreditTM by ACCME-accredited providers. APRNs who participate will receive a certificate of completion commensurate with the extent of their participation.
Date of original release: 23 February 2023. Date credits expire: 23 February 2024.
If you have any questions regarding credit please contact firstname.lastname@example.org
- Describe the prevalence and natural history of the rare lysosomal storage disorder alpha-mannosidosis (AM)
- Explain how diagnosis of AM may be approached, and describe the methods of disease identification
- Outline current and emerging management and treatment options for patients with AM
Faculty & Disclosures
Prof. Barbara K Burton
Prof. Barbara K Burton is a professor of paediatrics (genetics, genomics and metabolism) at the Northwestern University Feinberg School of Medicine and director of the Mucopolysaccharidoses (MPS) and Mucolipidoses Treatment Program at the Ann & Robert H. Lurie Children’s Hospital of Chicago. She is board certified in paediatrics, clinical genetics and clinical biochemical genetics. Her clinical and research interests are focused on inborn errors of metabolism and newborn screening. Prof. Burton is an investigator in numerous natural history studies and clinical trials of new therapies for various metabolic disorders, with a focus on lysosomal disorders, including MPS. She has published over 200 peer-reviewed articles, 50 chapters in books and is an editor of two textbooks. read more
Prof. Burton is active in professional organizations and is a past president of the Society for Inherited Metabolic Disorders and the Chicago Pediatric Society. She served for 4 years as a member of the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children, the federal advisory committee that makes recommendations regarding newborn screening in the US, and currently serves as chairman of the Newborn Screening Advisory Committee for the Illinois Department of Public Health. She is an emeritus member of the board of directors of the Greater Chicago chapter of March of Dimes, a nonprofit organization from whom she received a Lifetime Achievement Award in 2018. She is a member of the Scientific Advisory Board of the National MPS Society and serves on the medical advisory board of a number of other patient advocacy organizations.
Advisory board or panel: Agios, Alexion, Applied Therapeutics, Maze Therapeutics, Orchard Therapeutics. Consultant: Biomarin, Horizon, JCR Pharmaceuticals, Moderna, Passage Bio, Sanofi, Takeda, Ultragenyx. Grants/research support: Biomarin, Denali, Homology Medicines, JCR Pharmaceuticals, Sangamo, Ultragenyx. Speaker’s bureau: Biomarin, Horizon, Takeda.
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