Prof. Damara Ortiz provides an update on how prolonged survival in patients receiving enzyme replacement therapy (ERT) has altered the infantile-onset Pompe disease (IOPD) phenotype. She also considers the emerging clinical implications for effective clinical management strategies arising from improved understanding of IOPD as a multisystem disorder.1/3 Next Interview
Prof. Virginia Kimonis talks us through how improved clinical management and outcomes in the era of ERT has expanded our understanding of the phenotypic spectrum in LOPD. Now considered a multisystem disorder, Prof. Kimonis shares clinical insights on how the expanded LOPD phenotype may impact the treatment landscape and facilitate individualized care for patients living with LOPD.2/3 Next Interview
Prof. Loren Pena summarizes the current unmet needs surrounding existing enzyme replacement therapies (ERT) in Pompe disease, and provides an overview of new and emerging clinical management strategies, including gene therapy. She also reflects on how these latest developments may impact future clinical practice.3/3 Take CME Test
Overview & Learning Objectives
In this activity, leading experts explore the latest advances in our understanding of the emerging spectrum of Pompe disease in the ERT era, including expanding phenotypes and the multisystem nature of disease, as well as the impact on patients and their caregivers.
This activity has been jointly provided by Oakstone and touchIME. Oakstone Publishing is accredited by the ACCME to provide continuing medical education to physicians. read more
This activity has been designed to meet the educational needs of paediatricians, internal medicine physicians, cardiologists and neurologists involved in the multidisciplinary management of Pompe disease.
Oakstone Publishing has assessed conflict of interest with its faculty, authors, editors, and any individuals who were in a position to control the content of this CME activity. Any identified relevant conflicts of interest were resolved for fair balance and scientific objectivity of studies utilized in this activity. Oakstone Publishing’s planners, content reviewers, and editorial staff disclose no relevant commercial interests.
Prof. Damara Ortiz discloses: Grants/research support from Amicus, BioMarin, Sanofi-Genzyme and Takeda. Consultant/advisory boards for Amicus, Sanofi-Genzyme.
Prof. Virginia Kimonis discloses: Grants/research support from Sanofi-Genzyme. Consultant/advisory boards for Sanofi-Genzyme.
Prof. Loren Pena discloses: Grants/research support from Shire, Takeda. Consultant/advisory boards for Avexis, Orphazyme, Retrophin, Ultragenyx. Other financial or material support from Sanofi-Genzyme.
Walter Murray Yarbrough, MD has no financial interests/relationships or affiliations in relation to this activity.
Touch Medical Director
Alison Scott PhD has no financial interests/relationships or affiliations in relation to this activity.
Requirements for Successful Completion
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of Oakstone Publishing and touchIME. Oakstone Publishing is accredited by the ACCME to provide continuing medical education for physicians.
The European Union of Medical Specialists (UEMS) – European Accreditation Council for Continuing Medical Education (EACCME) has an agreement of mutual recognition of continuing medical education (CME) credit with the American Medical Association (AMA) European physicians interested in converting AMA PRA Category 1 Credit™ into European CME credit (ECMEC) should contact the UEMS (www.uems.eu).
Oakstone Publishing designates this enduring material for a maximum of 0.5 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
In order to receive credit for this activity, participants must review and complete the post-test and evaluation form. A score of 70% or higher is needed to obtain CME credit. Statements of credit are awarded upon successful completion of the post-test and evaluation form.
Date of original release: 30 September 2020. Date credits expire: 30 September 2021.
After watching this activity, participants should be better able to:
- Discuss how successful ERT has led to the new phenotype of infantile-onset Pompe disease
- Describe the multisystem disorders of late-onset Pompe disease
- Explain the approaches that are being used to address the unmet treatment needs for Pompe disease