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ForwardHoming in on Holistic Management of Late Onset PompeDisease: Contemporary Insights on Disease Monitoring, Treatment Selection and Switching, and Shared Decision-Making
A deeper understanding of Pompe disease has revealed a complex, multisystem disorder with a broad spectrum of manifestations, particularly in patients with late-onset Pompe disease (LOPD). Expanded newborn screening programs have further revealed the insidious nature of symptom onset before overt clinical signs appear. Emerging evidence supports the use of advanced imaging techniques — such as muscle ultrasound and magnetic resonance imaging for detecting early neuromuscular changes, even before abnormalities in creatine kinase or urine hexose tetrasaccharide levels are observed. Join our expert panel for a dynamic, case-based discussion on evolving insights into disease progression and how next-generation therapies are reshaping the management of LOPD. Gain practical strategies for diagnosis, monitoring, treatment selection, and shared decision-making in an era of expanding therapeutic options.
Target Audience
This initiative is intended for neuromuscular specialists, clinical/medical geneticists, neurologists, lysosomal disease specialists, genetic counselors, pediatricians, primary care/family medicine/general practice, as well as other HCPs involved in the diagnosis and management of LOPD, including nurses, pharmacists, and respiratory therapists.
Learning Objectives
Upon successful completion of this activity, participants should be better able to:
- Recognize contemporary unmet needs of those living with LOPD and their care partners
- Identify multi-modal, holistic assessment and diagnostic strategies, including emerging techniques, capable of identifying subclinical PD onset, disease activity, and/or progression on treatment
- Assess strategies, opportunities, and tools to individualize treatment decisions in LOPD, particularly for patients considering switching treatments
- Integrate principles aligned with shared decision-making that consider patient reported outcomes to optimize multidisciplinary LOPD management
Commercial Support
This activity is supported by an educational grant from Amicus Therapeutics, Inc.
Manchester Grand Hyatt San Diego
1 Market Pl, San Diego, CA 92101
All of the relevant financial relationships listed for these individuals have been mitigated.
Disclosures
None of the planners or faculty for this educational activity have relevant financial relationship(s) to disclose with ineligible companies whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients.
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.
In support of improving patient care, this activity has been planned and implemented by AKH Inc., Advancing Knowledge in Healthcare and Catalyst Medical Education, 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.
This activity was planned by and for the healthcare team, and learners will receive XX Interprofessional Continuing Education (IPCE) credit for learning and change.
Physicians
AKH Inc., Advancing Knowledge in Healthcare designates this live activity for a maximum of 1 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Nurses
Credit being awarded: 1 ANCC contact hours.
Pharmacists
AKH Inc., Advancing Knowledge in Healthcare designates this continuing education activity for 1 contact hours.
Respiratory Therapists
This educational activity is currently under submission to the American Association for Respiratory Care (AARC).
Genetic Counselor CEUs
To apply for National Society of Genetic Counselors Category 2 CEU, please go to https://www.nsgc.org/
The UEMS-EACCME® has had an agreement of mutual recognition of credits with the American Medical Association (AMA) for Live Educational Events and for E-Learning Materials since the year 2000. The agreement was renewed in 2022 for another 4 years.
The issue of territoriality is very important; both organizations are fully responsible for the activities taking place or organized within their remit. The UEMS-EACCME® is the central body for accrediting events in Europe and the AMA is the central body for recognition of CME credits in the USA.
Available Credit
- 1.00 ACPE Pharmacist
- 1.00 AMA PRA Category 1 Credit™
- 1.00 ANCC
- 1.00 Attendance