Fabry Disease Research Highlights: WORLDSymposium 2021
Jointly Provided by
Supported by an educational grant from Chiesi USA, Inc
Start date: April 30, 2021
End date: April 30, 2022
Estimated time to complete: 0.25 hours
Activity Description
At WORLDSymposium 2021, new information and data were presented on Fabry disease, a rare lysosomal storage disorder. This CME activity highlights the latest information about Fabry disease from WORLDSymposium 2021 and provides expert analysis of its clinical relevance for busy members of the care team in order to help them care for patients with this rare condition.
Activity Faculty
Derralynn Hughes, MD, DPhil, FDCP, FRCPath
Professor of Experimental Haematology
University College London
Clinical Director of Research and Innovation
Royal Free London NHS Foundation Trust
United Kingdom
Target Audience
This activity has been designed to meet the educational needs of physicians caring for individuals with Fabry disease. Other members of the care team may also participate.
Learning Objectives
After participating in the activity, learners should be better able to:
- Describe the latest research related to Fabry disease to better understand the condition and manage people with Fabry disease.
Accreditation and Credit Designation
In support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. 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
American Academy of CME, Inc., designates this enduring material for a maximum of 0.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Disclosure Statement
According to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relevant relationships with any commercial interests related to this activity. The existence of these interests or relationships is not viewed as implying bias or decreasing the value of the presentation. All educational materials are reviewed for fair balance, scientific objectivity and levels of evidence.
Disclosures are as follows:
Professor Hughes discloses the following financial relationships:
Advisory Board/Consultant: Amicus, Takeda, Sanofi, Idorsia, Freeline, Protalix, Biomarin Pharmaceuticals, Sangano
Promotional Speakers’ Bureau: Takeda, Sanofi, Amicus
Planners for this activity have no relevant financial relationships with any commercial interests.
This activity will review off-label or investigational information.
The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information.
Method of Participation
There are no fees to participate in the activity. Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the post-program assessment. Your certificate can be mailed to you in approximately 30 days.
Hardware/Software Requirements
Windows Requirements: • Operating system: Windows XP Service Pack 2 or later • Browser: Internet Explorer 7 or later, Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connection
Macintosh Requirements: • Operating system: Mac OS X v10.3 or later • Browser: Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connection
Privacy
For more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm. For more information about CheckRare’s privacy policy, please access https://checkrare.com/privacy/
Contact
For any questions, please contact: CEServices@academycme.org
Copyright
© 2021. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).
Fabry Disease Research Highlights: WORLDSymposium 2021
References
TOP FABRY DISEASE ABSTRACTS PUBLISHED FROM WORLDSYMPOSIUM 2021
Beck M, Ramaswami U, Hughes D, et al. Fabry Outcome Survey (FOS): Highlights from a 20-year patient registry of Fabry disease. Mol Genet Metab. 2021; 132 (suppl): S19.
Evison F, Franks D, Gallier S, Geberhiwot T. Evaluation of algorithms to identify patients with Fabry disease using routinely collected hospital activity data. Mol Genet Metab. 2021; 132 (suppl): S38.
Ueberall MA, Gloeckner B, Peceny M, Horlemann J. Development of a Fabry disease screening tool for chronic pain patients - step 1: Categorization based on phenotypic risk profiles. Mol Genet Metab. 2021; 132 (suppl): S107.
Jefferies JL, Spencer AK, Warnock DG, et al. Utilization of artificial intelligence to identify undiagnosed Fabry disease patients: Development of a validated machine learning model. Mol Genet Metab. 2021; 132 (suppl): S52.
Dutra-Clarke M, Tapia D, Curtin E, et al. Variable clinical features of patients with Fabry disease and outcome of enzyme replacement therapy. Mol Genet Metab. 2021; 132 (suppl): S36.
Linhart A, Dostalova G, Nicholls K, et al. Switching from agalsidase alfa to pegunigalsidase alfa to treat patients with Fabry disease: 1 year of treatment data from BRIDGE, a phase 3 open-label study. Mol Genet Metab. 2021; 132 (suppl): S65.
Augusto JB, Johner N, Shah D, et al. Proposed stages of cardiomyopathy in Fabry disease. Mol Genet Metab. 2021; 132 (suppl): S17.
Ganesh J, Goker-Alpan O, Jaggumantri S, Souberbielle B, Cockroft B. A phase I/II multicenter gene therapy clinical study for Fabry disease. Mol Genet Metab. 2021; 132 (suppl): S41-S42.
Thomas M, Nicholls K, Carnley B, et al. AVR-RD-01, an investigational lentiviral gene therapy for Fabry disease: Overview of clinical data from phase 1 and phase 2 studies. Mol Genet Metab. 2021; 132 (suppl): S106.
Laney DA, Germain DP, Oliveira JP, et al. Fabry disease and COVID-19: International expert recommendations for management based on real-world experience. Mol Genet Metab. 2021; 132 (suppl): S62.
Frey A, Trokan L, Vogler M, Hughes D, Gimona A. Measures to mitigate disruption due to the COVID-19 pandemic of the MODIFY phase 3 pivotal trial in patients with Fabry disease. Mol Genet Metab. 2021; 132 (suppl): S39-40.
CLINICAL TRIALS
To see list of clinical trials currently recruiting for Fabry disease, click here.
GUIDELINES
Germain DP, Foulihoux A, Decramer S, et al. Consensus recommendations for diagnosis, management and treatment of Fabry disease in paediatric patients. Clin Genet. 2019; 96: 107-117.
Hopkin RJ, Jefferies JL, Laney DA, et al. The management and treatment of children with Fabry disease: a United States‐based perspective. Mol Genet Metab. 2016; 117: 104‐113
Laney DA, Germain DP, Oliveira JP, et al. Fabry disease and COVID-19: international expert recommendations for management based on real-world experience. Clin Kidney J. 2020; 13: 913-925.
Jointly Provided by
Supported by an educational grant from Chiesi USA, Inc
Start date: April 30, 2021
End date: April 30, 2022
Estimated time to complete: 0.25 hours
Activity Description
At WORLDSymposium 2021, new information and data were presented on Fabry disease, a rare lysosomal storage disorder. This CME activity highlights the latest information about Fabry disease from WORLDSymposium 2021 and provides expert analysis of its clinical relevance for busy members of the care team in order to help them care for patients with this rare condition.
Activity Faculty
Derralynn Hughes, MD, DPhil, FDCP, FRCPath
Professor of Experimental Haematology
University College London
Clinical Director of Research and Innovation
Royal Free London NHS Foundation Trust
United Kingdom
Target Audience
This activity has been designed to meet the educational needs of physicians caring for individuals with Fabry disease. Other members of the care team may also participate.
Learning Objectives
After participating in the activity, learners should be better able to:
- Describe the latest research related to Fabry disease to better understand the condition and manage people with Fabry disease.
Accreditation and Credit Designation
In support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. 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
American Academy of CME, Inc., designates this enduring material for a maximum of 0.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Disclosure Statement
According to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relevant relationships with any commercial interests related to this activity. The existence of these interests or relationships is not viewed as implying bias or decreasing the value of the presentation. All educational materials are reviewed for fair balance, scientific objectivity and levels of evidence.
Disclosures are as follows:
Professor Hughes discloses the following financial relationships:
Advisory Board/Consultant: Amicus, Takeda, Sanofi, Idorsia, Freeline, Protalix, Biomarin Pharmaceuticals, Sangano
Promotional Speakers' Bureau: Takeda, Sanofi, Amicus
Planners for this activity have no relevant financial relationships with any commercial interests.
This activity will review off-label or investigational information.
The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information.
Method of Participation
There are no fees to participate in the activity. Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the post-program assessment. Your certificate can be mailed to you in approximately 30 days.
Hardware/Software Requirements
Windows Requirements: • Operating system: Windows XP Service Pack 2 or later • Browser: Internet Explorer 7 or later, Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connection
Macintosh Requirements: • Operating system: Mac OS X v10.3 or later • Browser: Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connection
Privacy
For more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm. For more information about CheckRare's privacy policy, please access https://checkrare.com/privacy/
Contact
For any questions, please contact: CEServices@academycme.org
Copyright
© 2021. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).
