In this online, self-learning activity:
Mucopolysaccharidoses (MPS) are a group of genetic diseases characterized by a deficiency of lysosomal enzymes responsible for the hydrolysis of glycosaminoglycans (GAGs), resulting in a variety of clinical manifestations in patients presenting with MPS. MPS has several subcategories, and MPS type I (MPS I) arises from the inheritance of an alteration in the IDUA gene, which encodes for alpha-L-iduronidase. Affecting an estimated one in 100,000 live births, MPS I is categorized as either attenuated MPS I (also known as Scheie or Hurler-Scheie syndromes) or severe MPS I with cognitive impairment (also known as Hurler syndrome).
Progressive in nature, MPS I is associated with multi-organ complications and sequelae. Patients exhibit a spectrum of clinical presentations, including facial deformities, organomegaly, cognitive impairments, upper airway obstructions, skeletal deformities, and cardiomyopathy. The burden of MPS I is considerable, with reports of caregivers contributing 51 hours per week on average to help patients perform daily activities of living. Quality of life for patients and their caregivers is significantly reduced with MPS I, affecting the social, emotional, and financial well-being of a family. It is reported that parents fear for their child’s delayed language acquisition, ability to fit in amongst peers and the society, fear of the expense for the necessary care, and fear for the death of a child from obstructive sleep apnea.
This learning activity has been designed to bring HCPs’ knowledge of the strategies for treatment and management of MPS I up to date and to improve their competence and performance in treating it.
The following HCPs: Pediatricians, neurologists, endocrinologists, genetic disease specialists; physician assistants, nurse practitioners, and pharmacists in the aforementioned areas of specialty; and any other HCPs with an interest in or who may clinically encounter patients with MPS I.
Commercial Support Disclosure: This program is supported by an educational grant from Sanofi.
This activity is free of charge.
Release Date: July 24, 2022 -- Expiration Date: July 24, 2024
Faculty: Paul Orchard, MD
Faculty introductions, disclosures |
MPS I introduction section
o Prevalence of the disease: Statistics
· Multi-organ pathogenesis · Disease manifestations · Early disease-detection
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MPS I treatment, healthcare burden, and barriers to care · HSCT o Timing o Optimizing outcomes o Posttransplant care · Enzyme replacement therapy o Treatment recommendations o Dosing, scheduling, and frequency o Safety and efficacy
o Reduced patient and caregiver quality of life o Need for ongoing treatment o Patient and caregiver education
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Conclusions, and best practice recap |
By the end of the session the participant will be able to:
ACCME Activity #202326570
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education through ScientiaCME. ScientiaCME is accredited by the ACCME to provide continuing medical education for physicians.
Credit Designation: ScientiaCME designates this educational activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™ toward the AMA Physician's Recognition Award. Physicians should only claim credit commensurate with the extent of their participation in the activity.
ABIM MOC Recognition Statement: Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.
ABIM MOC Credit Type: Medical Knowledge
ABP MOC Recognition Statement: Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1 MOC point in the American Board of Pediatrics' (ABP) Maintenance of Certification (MOC) program. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABP MOC credit.
ABP MOC Credit Type: Lifelong Learning and Self-Assessment
Physicians: For maintenance of certification (MOC) points, you must enter your board certification ID # and birth date correctly. It is the learner's responsibility to provide this information completely and accurately at the completion of the activity. Without providing it, the learner will NOT receive MOC points for this activity. By providing this data, you acknowledge that it will be shared with ACCME and the applicable certifying board. Please note: Not all activities on this site provide MOC points. If this activity does not specify that it provides MOC points in this section, then it does NOT provide MOC points. This activity provides MOC points only for ABIM.
Pharmacists
ScientiaCME is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 1.0 contact hours (0.1 CEUs) of continuing pharmacy education credit. Proof of participation will be posted to your NABP CPE profile within 4 to 6 weeks to participants who have successfully completed the post-test. Participants must participate in the entire presentation and complete the course evaluation to receive continuing pharmacy education credit. ACPE #0574-0000-22-038-H01-P. This is an knowledge (K)-type activity.
Pharmacists: You must enter your NABP # and birth date correctly so that proof of participation can be posted to your NABP CPE profile. It is the learner's responsibility to provide this information completely and accurately at the completion of the activity. Without providing it, the learner will NOT receive CPE credit for this activity.
Nurse Practitioners (NPs): The American Academy of Nurse Practitioners accepts AMA PRA Category 1 Credit(s)™ from organizations accredited by the ACCME. ScientiaCME will provide NPs who successfully complete each activity with a certificate of participation indicating that the activity was designated for AMA PRA Category 1 Credit(s)™.
Physician Assistants: The American Academy of Physician Assistants accepts AMA PRA Category 1 Credit(s)™ from organizations accredited by the ACCME.
As a provider of continuing medical education, it is the policy of ScientiaCME to ensure balance, independence, objectivity, and scientific rigor in all of its educational activities. In accordance with this policy, faculty and educational planners must disclose any significant relationships with commercial interests whose products or devices may be mentioned in faculty presentations, and any relationships with the commercial supporter of the activity. The intent of this disclosure is to provide the intended audience with information on which they can make their own judgments. Additionally, in the event a conflict of interest (COI) does exist, it is the policy of ScientiaCME to ensure that the COI is resolved in order to ensure the integrity of the CME activity. For this CME activity, any COI has been resolved thru content review by ScientiaCME.
Faculty Disclosure: Paul Orchard, MD, has received financial compensation for consulting for Orchard Therapeutics, Neurogene, and Avrobio and has had clinical trials and research supported by Genzyme/Sanofi, Bluebirdbio, and Immusoft.
Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, President of ScientiaCME, has no relevant financial disclosures.
Faculty will discuss off-label uses of a commercial product.
All relevant financial relationships have been mitigated.
ScientiaCME 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 CE activity, including faculty, planners, reviewers or others are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity.
Commercial Support Disclosure: This program is supported by an educational grant from Sanofi.
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