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The Future of Muscular Dystrophy Management: Updates for Limb Girdle Muscular Dystrophy

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The Future of Muscular Dystrophy Management: Updates for Limb Girdle Muscular Dystrophy

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15 minutes
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  • Overview

    Limb-girdle dystrophy (LGMD) is a group of inherited neuromuscular diseases with at least 34 variants, all affecting muscles of the limb-girdle region. Because LGMD is so variable, it can be hard to detect and there are no FDA-approved treatments currently. Advances in gene transfer therapy have been slow to emerge. However, helping clinicians detect LGMD and know the next best steps is critical to effective management of LGMD, along with identifying patients for clinical trials.

  • Disclosure of Conflicts of Interest

    In accordance with the ACCME Standards for Integrity and Independence in Accredited Continuing Education, The France Foundation (TFF) requires that individuals in a position to control the content of an educational activity disclose all relevant financial relationships with any commercial entity. TFF resolves all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all its educational programs. Furthermore, TFF seeks to verify that all scientific research referred to, reported, or used in a CME/CE activity conforms to the generally accepted standards of experimental design, data collection, and analysis. TFF is committed to providing learners with high-quality CME/CE activities that promote improvements in health care and not those of a commercial interest.

    Planning committee members, faculty, reviewers, and activity staff have disclosed the following relevant financial relationships. All relevant financial relationships listed have been mitigated.

    FACULTY/PLANNERS:
    John Brandsema, MD
    Non-CE Consulting: AveXis/Novartis, Biogen, Dyne, Fibrogen, Genentech, Momenta/Janssen, NS Pharma, Pfizer, PTC Therapeutics, Sarepta, Scholar Rock, and Takeda
    Contract Research: Alexion, Astellas, AveXis/Novartis, Biogen, CSL Behring, Cytokinetics, Dyne, Fibrogen, Genentech, Pfizer, PTC Therapeutics, and Sarepta

    Vamshi Rao, MD
    Advisory Board or Panel: Biogen, NSPharma, Novartis, PTC therapeutics, RegenxBio, Sarepta, and Scholar Rock
    Consultant: Delsys, Genetech/Roche, Novartis, and PTC therapeutics
    Grants/Research Support: NSPharma, RegenxBio, and Sarepta
    Speakers Bureau or other Promotional Education: Biogen and Genetech/Roche

    PLANNER
    Kittredge White, BSc, MA, PA-C
    No relevant financial disclosures

    ACTIVITY STAFF
    Jaspreet Chahal, MS, MA, No relevant financial disclosures
    Heather Tarbox, MPH, No relevant financial disclosures
    Janis Vajdos, MS, No relevant financial disclosures

  • Target Audience

    Clinicians who have a role in the management of patients with LGMD, including pediatric and developmental neurologists, neuromuscular specialists, pediatricians, geneticists, genetic counsellors, physical and occupational therapists, nurse practitioners, and physician associates.

  • Learning Objectives

    • Identify existing LGMD genetic testing programs and determine appropriate patient pathways for receiving specialized care
    • Recognize the importance of early diagnosis in LGMD and the consequences of missed or delayed diagnosis
    • Assess how ongoing developments in clinical research with gene therapy may help address current limitations in the management of LGMD
  • Accreditation and Credit Designation Statements

    Accreditation Statement


    In support of improving patient care, The France Foundation 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 health care team. 

    Physician Credit Designation

    The France Foundation designates this enduring activity for a maximum of 0.25 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

    Nurses

    The France Foundation designates this activity for 0.25 contact hours.

    PHYSICIAN ASSISTANTS

    The France Foundation 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 0.25 AAPA Category 1 CME credits. Approval is valid until June 29, 2024. PAs should only claim credit commensurate with the extent of their participation.

    All other health care professionals completing this course will be issued a statement of participation.

  • Disclaimer

    Disclaimer
    The France Foundation presents this information for educational purposes only. The content is provided solely by faculty who have been selected because of recognized expertise in their field. Participants have the professional responsibility to ensure that products are prescribed and used appropriately on the basis of their own clinical judgment and accepted standards of care. The France Foundation and the commercial supporter(s) assume no liability for the information herein.

  • Provider(s)/Educational Partner(s)


    This activity is provided by The France Foundation.

  • Commercial Support

    This activity is supported by an independent medical educational grant from Sarepta Therapeutics, Inc.

  • System Requirements

    • Supported Browsers (2 most recent versions):
      • Google Chrome for Windows, Mac OS, iOS, and Android
      • Apple Safari for Mac OS and iOS
      • Mozilla Firefox for Windows, Mac OS, iOS, and Android
      • Microsoft Edge for Windows
    • Recommended Internet Speed: 5Mbps+

  • Publication Dates

    Release Date:

    Expiration Date:

Facebook Comments

Recommended
Details
Presenters
Related
Comments
  • Overview

    Limb-girdle dystrophy (LGMD) is a group of inherited neuromuscular diseases with at least 34 variants, all affecting muscles of the limb-girdle region. Because LGMD is so variable, it can be hard to detect and there are no FDA-approved treatments currently. Advances in gene transfer therapy have been slow to emerge. However, helping clinicians detect LGMD and know the next best steps is critical to effective management of LGMD, along with identifying patients for clinical trials.

  • Disclosure of Conflicts of Interest

    In accordance with the ACCME Standards for Integrity and Independence in Accredited Continuing Education, The France Foundation (TFF) requires that individuals in a position to control the content of an educational activity disclose all relevant financial relationships with any commercial entity. TFF resolves all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all its educational programs. Furthermore, TFF seeks to verify that all scientific research referred to, reported, or used in a CME/CE activity conforms to the generally accepted standards of experimental design, data collection, and analysis. TFF is committed to providing learners with high-quality CME/CE activities that promote improvements in health care and not those of a commercial interest.

    Planning committee members, faculty, reviewers, and activity staff have disclosed the following relevant financial relationships. All relevant financial relationships listed have been mitigated.

    FACULTY/PLANNERS:
    John Brandsema, MD
    Non-CE Consulting: AveXis/Novartis, Biogen, Dyne, Fibrogen, Genentech, Momenta/Janssen, NS Pharma, Pfizer, PTC Therapeutics, Sarepta, Scholar Rock, and Takeda
    Contract Research: Alexion, Astellas, AveXis/Novartis, Biogen, CSL Behring, Cytokinetics, Dyne, Fibrogen, Genentech, Pfizer, PTC Therapeutics, and Sarepta

    Vamshi Rao, MD
    Advisory Board or Panel: Biogen, NSPharma, Novartis, PTC therapeutics, RegenxBio, Sarepta, and Scholar Rock
    Consultant: Delsys, Genetech/Roche, Novartis, and PTC therapeutics
    Grants/Research Support: NSPharma, RegenxBio, and Sarepta
    Speakers Bureau or other Promotional Education: Biogen and Genetech/Roche

    PLANNER
    Kittredge White, BSc, MA, PA-C
    No relevant financial disclosures

    ACTIVITY STAFF
    Jaspreet Chahal, MS, MA, No relevant financial disclosures
    Heather Tarbox, MPH, No relevant financial disclosures
    Janis Vajdos, MS, No relevant financial disclosures

  • Target Audience

    Clinicians who have a role in the management of patients with LGMD, including pediatric and developmental neurologists, neuromuscular specialists, pediatricians, geneticists, genetic counsellors, physical and occupational therapists, nurse practitioners, and physician associates.

  • Learning Objectives

    • Identify existing LGMD genetic testing programs and determine appropriate patient pathways for receiving specialized care
    • Recognize the importance of early diagnosis in LGMD and the consequences of missed or delayed diagnosis
    • Assess how ongoing developments in clinical research with gene therapy may help address current limitations in the management of LGMD
  • Accreditation and Credit Designation Statements

    Accreditation Statement


    In support of improving patient care, The France Foundation 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 health care team. 

    Physician Credit Designation

    The France Foundation designates this enduring activity for a maximum of 0.25 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

    Nurses

    The France Foundation designates this activity for 0.25 contact hours.

    PHYSICIAN ASSISTANTS

    The France Foundation 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 0.25 AAPA Category 1 CME credits. Approval is valid until June 29, 2024. PAs should only claim credit commensurate with the extent of their participation.

    All other health care professionals completing this course will be issued a statement of participation.

  • Disclaimer

    Disclaimer
    The France Foundation presents this information for educational purposes only. The content is provided solely by faculty who have been selected because of recognized expertise in their field. Participants have the professional responsibility to ensure that products are prescribed and used appropriately on the basis of their own clinical judgment and accepted standards of care. The France Foundation and the commercial supporter(s) assume no liability for the information herein.

  • Provider(s)/Educational Partner(s)


    This activity is provided by The France Foundation.

  • Commercial Support

    This activity is supported by an independent medical educational grant from Sarepta Therapeutics, Inc.

  • System Requirements

    • Supported Browsers (2 most recent versions):
      • Google Chrome for Windows, Mac OS, iOS, and Android
      • Apple Safari for Mac OS and iOS
      • Mozilla Firefox for Windows, Mac OS, iOS, and Android
      • Microsoft Edge for Windows
    • Recommended Internet Speed: 5Mbps+

  • Publication Dates

    Release Date:

    Expiration Date:

Facebook Comments

Schedule18 Jun 2024