This activity is supported by an independent education grant from Amicus. This online education program has been designed solely for healthcare professionals in the USA. The information is this program is not suitable for any other country.

Join leading experts Dr Barbara Burton and Dr Tahseen Mozaffar for this accredited virtual webinar. This 45-minute expert-led webinar is designed to build clinician confidence in managing adults with Late-Onset Pompe Disease (LOPD). Through an evolving case of a single adult patient followed across multiple clinical visits, faculty demonstrate how experienced clinicians interpret subtle monitoring changes to distinguish apparent disease stability from progressive decline.

Participants will analyze longitudinal motor, respiratory, imaging, and patient-reported outcomes data across three distinct clinical timepoints to identify early indicators of loss of therapeutic benefit. By evaluating these evolving metrics, attendees will learn when management strategies should be reconsidered and how to effectively incorporate multidisciplinary care approaches.

Session Highlights

• Longitudinal Case Tracking: Examine a single adult LOPD patient journey across three clinical timepoints to observe real-world disease evolution and decision-making.
• Suboptimal Response Recognition: Analyze motor and respiratory assessment trends to spot early functional decline despite apparent clinical stability.
• Multimodal Data Integration: Combine respiratory metrics, imaging findings, and patient-reported outcomes to detect subtle, subclinical progression.
• Management Reassessment: Evaluate when to reconsider current treatment strategies and explore timing for multidisciplinary care interventions.

Who Should Watch

This program is designed for HCPs involved in the monitoring and longitudinal management of adults with LOPD, including:

• Neuromuscular specialists
• Neurologists
• Metabolic disease specialists

Faculty

Barbara K. Burton, Professor Emeritus, is a medical geneticist and faculty member at Northwestern University Feinberg School of Medicine. Triple-board-certified in pediatrics, clinical genetics, and clinical biochemical genetics, she earned her medical degree from Northwestern University and completed her pediatric residency and genetics fellowship at Children’s Memorial Hospital. Dr. Burton’s clinical practice and research focus on the diagnosis, management, and treatment of inherited metabolic disorders and rare genetic conditions. She possesses specialized expertise in disorders identified through newborn screening—such as phenylketonuria (PKU)—as well as lysosomal storage disorders, and actively serves as an investigator in clinical trials evaluating novel therapies for rare diseases.

Dr. Tahseen Mozaffar, MD, is a board-certified neurologist and Director of the UCI Health ALS & Neuromuscular Center. He earned his medical degree from Aga Khan University, followed by a neurology residency at Barnes-Jewish Hospital and a neuromuscular fellowship at Washington University in St. Louis. Specializing in amyotrophic lateral sclerosis (ALS), myasthenia gravis, Pompe disease, and rare muscular dystrophies, Dr. Mozaffar leads extensive clinical and translational research as the principal investigator for over a dozen trials and lead UCI investigator for the NINDS-supported NeuroNEXT network. A prolific researcher with more than 175 peer-reviewed publications, he serves as an NIH reviewer and consultant for global pharmaceutical companies on trial design and safety monitoring in rare neuromuscular diseases.

Disclosures

Partners for Advancing Clinical Education (Partners) requires every individual in a position to control educational content to disclose all financial relationships with ineligible companies that have occurred within the past 24 months. Ineligible companies are organizations whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients. All relevant financial relationships are mitigated according to Partners policies.

Dr Barbara Burton has the following relevant financial relationships: BioMarin: Speaker, consultant; Chiesi: Speaker; Denali: Speaker, consultant; JCR Pharma: Consultant; Moderna: Consultant.

Dr Tahseen Mozaffar has the following relevant financial relationships: Amicus: Consulting, Research Funding; Aro Bio: Research Funding; Denali: Consulting, Research Funding; Regeneron: Consulting; Sanofi: Consulting, Research Funding; Shionogi: Consulting and Research Funding.

Joint Accreditation Statement

In support of improving patient care, this activity has been planned and implemented by Partners for Advancing Clinical Education (Partners) and MedAll. Partners 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.

Physician Continuing Education

Partners designates this live activity for a maximum of 0.75 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nursing Continuing Professional Development

The maximum number of hours awarded for this Nursing Continuing Professional Development activity is 0.75 ANCC contact hours.

PA Continuing Medical Education

Partners has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with AAPA CME Criteria. This activity is designated for 0.75 AAPA Category 1 CME credits. PAs should only claim credit commensurate with the extent of their participation

Pharmacy Continuing Education

Partners designates this continuing education activity for 0.75 contact hour(s) (0.75 CEUs) of the Accreditation Council for Pharmacy Education.

(Universal Activity Number - JA4008073-9999-26-110-L01-P)

Type of Activity: Application

For Pharmacists: Upon completion of the online evaluation, your credit will be submitted to CPE Monitor. Pharmacists have up to thirty (30) days to complete the evaluation and claim credit. Please check your NABP account within thirty (30) days to make sure the credit has posted.

Disclosure of Unlabeled Use

This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications. The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

Disclaimer

Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

Fee Information

There is no fee for this educational activity.