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ForwardThe detection and diagnosis of Niemann-Pick type C
This case-based educational program is designed to improve clinician competence in the early recognition and diagnosis ofNiemann–Pick disease type C (NPC). Through two interactive virtual patient simulations based on real-world diagnosticscenarios, participants will learn to identify age-dependent clinical red flags, differentiate NPC from common neurologicand psychiatric mimics, apply contemporary diagnostic pathways, interpret biomarker and genetic testing results, andrecognize the diagnostic significance of ocular motor abnormalities such as vertical supranuclear saccade palsy. The activity aims to reduce diagnostic delay and support earlier referral and multidisciplinary evaluation of children andadolescents with suspected NPC.
Target Audience
This activity is designed for a physician audience of Neurologists, Pediatric Neurologists, Pediatricians, Medical Geneticists, and Psychiatrists.
Learning Objectives
- Recognize age-dependent NPC red flags in early childhood, including unexplained splenomegaly, developmental concerns, progressive gait or coordination changes, and cataplexy-like events.
- Differentiate NPC from common pediatric mimics.
- Apply current diagnostic pathways for suspected pediatric NPC, including appropriate selection and sequencing of biomarker testing and confirmatory genetic evaluation to reduce time to diagnosis.
- Evaluate and interpret challenging diagnostic results in pediatric NPC.
- Implement practice changes to enable earlier identification and referral, including when to escalate testing, coordinate multidisciplinary evaluation, and initiate longitudinal monitoring to improve outcomes.
- Recognize the diagnostic significance of ocular motor abnormalities and understand when their presence should prompt NPC evaluation.
Commercial Support
This activity is supported from an independent medical education grant from Zevra Therapeutics.
DISCLOSURES | ||
Name | Relationship | Commercial Interest |
Barbara Burton MD (faculty) | Speaker | Biomarin, Zevra |
Consultant | Alltrna, Applied Therapeutics, Chiesi, Denali, JCR Pharma, Moderna, Ultragenyx, Uniqure | |
Caroline Hastings MD (faculty) | Consultant | Azafaros, Zevra, IntraBio, Cyclo Therapeutics |
Dorothy Caputo, MA, BSN, RN - President | N/A | Nothing to disclose |
Rebecca Lakowski, RN (planner/reviewer) | N/A | Nothing to disclose |
AKH Inc Staff and Planners | N/A | Nothing to disclose |
Orakle LLC Staff and Planners | N/A | Nothing to disclose |
All of the relevant financial relationships listed for these individuals have been mitigated. | ||
Disclosures
It is the policy of AKH Inc. to ensure independence, balance, objectivity, scientific rigor, and integrity in all of its continuing education activities. The author must disclose to the participants any significant relationships with ineligible companies whose products or devices may be mentioned in the activity or with the commercial supporter of this continuing education activity. Identified conflicts of interest are mitigated by AKH prior to accreditation of the activity. AKH planners and reviewers have no relevant financial relationships to disclose.
Disclosure of Unlabeled Use and Investigational Product
This educational activity may include discussion of uses of agents that are investigational and/or unapproved by the FDA. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.
Disclaimer
This course is designed solely to provide the healthcare professional with information to assist in his/her practice and professional development and is not to be considered a diagnostic tool to replace professional advice or treatment. The course serves as a general guide to the healthcare professional, and therefore, cannot be considered as giving legal, nursing, medical, or other professional advice in specific cases. AKH Inc. specifically disclaim responsibility for any adverse consequences resulting directly or indirectly from information in the course, for undetected error, or through participants misunderstanding of the content. If you would like to opt out from future communications from AKH please send an email to [email protected] with your information with "Opt Out" in the subject line.
Barbara Burton
Caroline Hastings, MD
In support of improving patient care, this activity has been planned and implemented by AKH Inc., Advancing Knowledge in Healthcare and Orakle LLC. AKH Inc., Advancing Knowledge in Healthcare is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.
Physicians
AKH Inc., Advancing Knowledge in Healthcare designates this enduring material for a maximum of .50 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Available Credit
- 1.00 AMA PRA Category 1 Credit™
Physicians
AKH Inc., Advancing Knowledge in Healthcare designates this Enduring Material for a maximum of 1.00 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. - 1.00 Attendance
Credits: 1.00
- 1.00 AMA PRA Category 1 Credit™ Non-MD
Non-Physicians Seeking AMA Credit
AKH Inc., Advancing Knowledge in Healthcare designates this Enduring Material for a maximum of 1.00 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.