STATEMENT OF NEED
Acid sphingomyelinase deficiency (ASMD) is a rare, hereditary lysosomal storage disease with multisystemic manifestations, including splenomegaly, hepatomegaly, interstitial lung disease, dyslipidemia, bone marrow and lymph node involvement, and sometimes neurological and peripheral nerve involvement. While the most severe form of ASMD, infantile neurovisceral ASMD, results in death in early childhood, chronic forms of ASMD have more prolonged survival but are associated with substantial morbidity and reduced life expectancy. Outcomes for adults with chronic ASMD are affected by diagnostic delays that in turn delay monitoring and management of ASMD manifestations (Wasserstein et al, 2019). Furthermore, while treatment of ASMD has until now been solely supportive in nature, an acid sphingomyelinase (ASM) enzyme replacement therapy has recently been approved, presenting the first opportunity to address the pathology underlying this condition (US Food & Drug Administration, 2022). This activity chaired by Dr. Neal Weinreb, Voluntary Associate Professor for Human Genetics at the University of Miami Miller School of Medicine, will provide expert perspectives on current challenges and new opportunities in the management of ASMD in adults.
Metabolic disease specialists, medical geneticists, pulmonologists, hepatologists, gastroenterologists, neurologists, hematologists, primary care physicians, nurse practitioners, physician assistants, nurses, pharmacists, and other health care professionals involved in the treatment of patients with acid sphingomyelinase deficiency (ASMD).
Neal Weinreb, MD
Voluntary Associate Professor for Human Genetics
University of Miami
Miller School of Medicine
There is no fee to participate in or claim CME/NCPD credit for this activity.
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i3 Health designates this enduring activity for a maximum of 1.25 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.25 Medical Knowledge MOC points in the American Board of Internal Medicine’s (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider’s responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.
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American Academy of Physician Assistants (AAPA) accepts certificates of participation for educational activities approved for AMA PRA Category 1 Credit™ from organizations accredited by ACCME. Physician assistants may receive a maximum of 1.25 hours of Category 1 credit for completing this program.
The American Academy of Nurse Practitioners Certification Program (AANPCP) accepts AMA PRA Category 1 Credit™ from organizations accredited by the ACCME. Individuals are responsible for checking with the AANPCP for further guidelines.
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Provider approved by the California Board of Registered Nursing, Provider Number 15824, for 1.25 contact hours.
DISCLOSURE OF RELEVANT FINANCIAL INFORMATION WITH INELIGIBLE COMPANIES
i3 Health endorses the standards of the ACCME and ANCC that require everyone in a position to control the content of a CME/NCPD activity to disclose all financial relationships with ineligible companies that are related to the content of the CME/NCPD activity. CME/NCPD activities must be balanced, independent of commercial bias, and promote improvements or quality in health care. All recommendations involving clinical medicine must be based on evidence accepted within the medical profession.
A conflict of interest is created when an individual has an opportunity to affect CME/NCPD content about products or services of an ineligible company with which he/she has a financial relationship, which therefore may bias their opinions and teaching. This may include receiving a salary, royalty, intellectual property rights, consulting fee, honoraria, stocks, or other financial benefits.
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Relevant financial relationships exist between the following individuals and ineligible companies:
The i3 Health planners, reviewers, and managers have nothing to disclose.
Neal Weinreb, MD, discloses that he has served on an advisory board or panel for Avrobio, Pfizer, Sanofi, and Takeda; that he has served on the speaker's bureau for Sanofi; and that he has served as a consultant for Pfizer and Sanofi.
i3 Health has mitigated all relevant financial relationships.
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UNAPPROVED USE DISCLOSURE
i3 Health requires CME/NCPD faculty (speakers) to disclose to attendees when products or procedures being discussed are off-label, unlabeled, experimental, and/or investigational (not FDA approved), as well as any limitations on the information that is presented, such as data that are preliminary or that represent ongoing research, interim analyses, and/or unsupported opinion. Faculty may discuss information about pharmaceutical agents that is outside of U.S. Food and Drug Administration approved labeling.
This information is intended solely for continuing medical education and is not intended to promote off-label use of these medications. If you have questions, contact the medical affairs department of the manufacturer for the most recent prescribing information.
The information provided at this CME/NCPD activity is for continuing education purposes only and is not meant to substitute for the independent medical/clinical judgment of a health care provider relative to diagnostic and treatment options of a specific patient’s medical condition.
This activity is supported by an independent medical educational grant from Sanofi.