Improving outcomes in the diagnosis of Niemann-Pick disease type C (NPC)
Release Date: August 31, 2025
Expiration Date: August 31, 2026
Activity Overview
This activity prepares healthcare teams to identify, diagnose, and coordinate care for patients with suspected NPC - which is critical for timely referrals and interdisciplinary management. Case-based modules simulate real-world diagnostic challenges, promoting the critical thinking and multidisciplinary collaboration necessary to recognize and manage complex, rare diseases like NPC.
Target Audience
This activity is designed for an audience of Physicians.
Learning Objectives
Upon completion of the educational activity, participants should be able to:
- Identify classic and atypical clinical presentations of NPC across age groups
- Differentiate NPC from primary psychiatric and neurodevelopmental disorders
- Apply a multi-modal diagnostic strategy integrating clinical, biochemical, and molecular data
Expiration Date: August 31, 2026
Activity Overview
This activity prepares healthcare teams to identify, diagnose, and coordinate care for patients with suspected NPC - which is critical for timely referrals and interdisciplinary management. Case-based modules simulate real-world diagnostic challenges, promoting the critical thinking and multidisciplinary collaboration necessary to recognize and manage complex, rare diseases like NPC.
Target Audience
This activity is designed for an audience of Physicians.
Learning Objectives
Upon completion of the educational activity, participants should be able to:
- Identify classic and atypical clinical presentations of NPC across age groups
- Differentiate NPC from primary psychiatric and neurodevelopmental disorders
- Apply a multi-modal diagnostic strategy integrating clinical, biochemical, and molecular data
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Criteria for Success
To obtain a certificate of completion, a score of 70% or better on the post-test is required. Please proceed with the activity until you have successfully completed this program, answered all test questions, completed the post-test and evaluation, and have received a digital copy of your certificate. You must participate in the entire activity to receive credit. There is no fee to participate in this activity. If you have questions about this activity, please contact AKH Inc. at ketrea@akhcme.com. https://akhinc.formstack.com/forms/250175e2
To obtain a certificate of completion, a score of 70% or better on the post-test is required. Please proceed with the activity until you have successfully completed this program, answered all test questions, completed the post-test and evaluation, and have received a digital copy of your certificate. You must participate in the entire activity to receive credit. There is no fee to participate in this activity. If you have questions about this activity, please contact AKH Inc. at ketrea@akhcme.com. https://akhinc.formstack.com/forms/250175e2
CE credit provided by AKH Inc., Advancing Knowledge in Healthcare.
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.
Walla Al-Hertani, MD, MSc, FRCPC, FCCMG, FACMG (faculty)
Tarekegn Hiwot MD, Msc, PhD (faculty)
Lucy: A young girl presents with developmental regression.
George: A 34-year-old man presents with treatment-resistant psychosis and progressive gait instability.
Physicians
AKH Inc., Advancing Knowledge in Healthcare designates this enduring 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.
Commercial Support
This activity is supported from an independent medical education grant from Zevra Therapeutics.
Disclosures
Walla Al-Hertani, MD, MSc, FRCPC, FCCMG, FACMG (faculty)
Advisor: Agyany Pharma, Amicus Therapeutics, Azafaros, Intrabio, Cyclo Therapeutics, HCU Network America (HCUNA), Kriya Therapeutics, National Niemann-Pick disease Foundation (NNPDF), Takeda Pharmaceuticals, Ultragenyx, Zevra Therapeutics.
Consultant: Beam Therapeutics.Researcher: Denali Therapeutics (Genetics Evaluation), Sanofi - International Collaborative Gaucher Group (ICGG) Registry.
Tarekegn Hiwot MD, Msc, PhD (faculty)
Nothing to disclose
Dorothy Caputo, MA, BSN, RN - President
Nothing to disclose
Michele Bielarski, RN (planner/reviewer)
Nothing to disclose
AKH Inc Staff and Planners
Nothing to disclose
Orakle LLC Staff and Planners
Nothing to disclose
All of the relevant financial relationships listed for these individuals have been mitigated.
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 optout@akhcme.com with your information with "Opt Out" in the subject line.
AKH Inc., Advancing Knowledge in Healthcare designates this enduring 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.
Commercial Support
This activity is supported from an independent medical education grant from Zevra Therapeutics.
Disclosures
Walla Al-Hertani, MD, MSc, FRCPC, FCCMG, FACMG (faculty)
Advisor: Agyany Pharma, Amicus Therapeutics, Azafaros, Intrabio, Cyclo Therapeutics, HCU Network America (HCUNA), Kriya Therapeutics, National Niemann-Pick disease Foundation (NNPDF), Takeda Pharmaceuticals, Ultragenyx, Zevra Therapeutics.
Consultant: Beam Therapeutics.Researcher: Denali Therapeutics (Genetics Evaluation), Sanofi - International Collaborative Gaucher Group (ICGG) Registry.
Tarekegn Hiwot MD, Msc, PhD (faculty)
Nothing to disclose
Dorothy Caputo, MA, BSN, RN - President
Nothing to disclose
Michele Bielarski, RN (planner/reviewer)
Nothing to disclose
AKH Inc Staff and Planners
Nothing to disclose
Orakle LLC Staff and Planners
Nothing to disclose
All of the relevant financial relationships listed for these individuals have been mitigated.
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 optout@akhcme.com with your information with "Opt Out" in the subject line.
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