ScientiaCME Safe and sound: empowering professionals with insights into the identification and management of alpha-mannosidosis
In this online, self-learning activity:
Alpha-mannosidosis (AM) is a rare recessive lysosomal storage disorder characterized by immune deficiency, facial and skeletal abnormalities, hearing impairment, and intellectual disability. It is caused by mutations in the MAN2B1 gene, leading to deficiency in alpha-mannosidase, a lysosomal enzyme involved in the oligosaccharide degradation pathway. While the exact prevalence of AM in the US is unknown, it has an estimated incidence of 1 in 500,000 live births worldwide. Laboratory indicators of AM often appear shortly after birth, followed by progression of clinical manifestations. While symptoms of AM vary considerably in individual presentations, three clinical subtypes have been identified (mild, moderate, and severe) and are used to guide disease prognosis and management. The current diagnostic algorithm for AM focuses on measuring alpha-mannosidase activity in leukocytes using colorimetry or fluorimetry. An alpha-mannosidase activity level of under 5% suggests AM, and a diagnosis is then confirmed with genetic sequencing.
Cost: Free
View Offer chevron_rightKey Features
Free CME Course
Topics Covered
Faculty introduction, disclosures
Introductory content:
Epidemiology: statistics and burden
Natural history and clinical presentations
Best practices in diagnosis: colorimetry, fluorimetry, genetic sequencing
Severity classification
Treatment of AM
· Disease management
o Supportive care for symptom management
o Hematopoietic stem cell transplantation
o ERT: safety and efficacy from registry and clinical trial data
– ADAs in ERTs for other conditions
– ADAs in AM ERT
· Future directions
· Patient case(s)
Summary, conclusions, and best practice recap
Learning Objectives
By the end of the session the participant will be able to:
Recall the natural history, diagnostic criteria, burden of illness, clinical presentations, and classification by severity of AM.
Identify currently available treatment options for patients with AM.
Summarize safety and efficacy data of ERT for patients with AM.
Formulate a treatment plan for a patient with AM.
Target Audience
HCPs including but not limited to: metabolic disease specialists, medical geneticists, pediatricians, and primary care providers; physician associates, nurse practitioners, and nurses who practice in the aforementioned areas of specialty; and any other HCPs with an interest in or who clinically encounter patients with inherited metabolic disorders.
Additional credit info
ACCME Activity #202888461
ScientiaCME is accredited by the Accreditation Council for Continuing Medical Education (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.
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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 1.0 MOC points 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 and ABP.
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 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 #0574-0000-25-001-H01-P. This is an Application (A)-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.
Nurses: The American Nurses Credentialing Center (ANCC) accepts AMA PRA Category 1 Credit(s)™ from organizations accredited by the ACCME. This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™.
Physician Assistants: The American Academy of Physician Assistants (AAPA) accepts AMA PRA Category 1 Credit™ assigned by organizations accredited by the ACCME as satisfying Category 1 CME for National Commission on Certification of Physician Assistants (NCCPA) national certification maintenance. This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™.
Nurse Practitioners: The American Academy of Nurse Practitioners Certification Board (AANPCB) states that continuing education providers accredited by the ACCME may provide acceptable, accredited Advanced Practice Provider content. This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™.