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CME / CE

A Review of Rare Conditions Across the Lifespan: Pediatric Neuromuscular Disorders

  • Authors: Claudia A. Chiriboga, MD, MPH, FAAN; Perry B. Shieh, MD, PhD; John F. Brandsema, MD; Diana P. Castro, MD; Emma Ciafaloni, MD; Basil T. Darras, MD; Bakri H. Elsheikh, MBBS
  • CME / CE Released: 10/11/2022
  • Valid for credit through: 10/11/2023
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  • Credits Available

    Physicians - maximum of 2.25 AMA PRA Category 1 Credit(s)™

    Nurses - 2.25 ANCC Contact Hour(s) (1 contact hours are in the area of pharmacology)

    You Are Eligible For

    • Letter of Completion

Target Audience and Goal Statement

This initiative is intended for neurologists, nurse practitioners (NPs), and nurses involved in the care of patients with rare neuromuscular diseases.

The goal of this activity is for learners to be better able to manage patients with pediatric neuromuscular disorders.

Upon completion of this activity, participants will:

  • Have increased knowledge regarding the
    • Pathophysiologic basis of muscular dystrophies
    • Strategies to reduce the time to diagnosis of muscular dystrophies
    • Clinical data for new and emerging therapies for the management of muscular dystrophies
    • Strategies to support patient access to timely care following a diagnosis of spinal muscular atrophy
    • Clinical trial data for therapies used for the management of spinal muscular atrophy
  • Have greater competence related to
    • Development of a comprehensive treatment plan for the management of adults with spinal muscular atrophy


Disclosures

Medscape, LLC 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 for anyone with the ability to control the content of this educational activity are listed below and have been mitigated. Others involved in the planning of this activity have no relevant financial relationships.

Disclosures for additional planners can be found here.


Session Co-Chair

  • Claudia A. Chiriboga, MD, MPH, FAAN

    Professor of Neurology and Pediatrics
    Division of Pediatric Neurology
    Columbia University Medical Center
    New York, New York

     

    Disclosures

    Claudia A. Chiriboga, MD, MPH, FAAN, has the following relevant financial relationships:
    Consultant or advisor for: Genentech; Novartis; PTC; Roche
    Speaker or member of speakers bureau for: Biogen Colombia; Genentech; Roche
    Research funding from: Avexis/Novartis; Biogen; Roche

  • Perry B. Shieh, MD, PhD

    Professor of Neurology
    David Geffen School of Medicine
    University of California
    Los Angeles, California

    Disclosures

    Perry B. Shieh, MD, PhD, has the following relevant financial relationships:
    Consultant or advisor for: Alexion; Biogen; Genentech; Novartis Gene Therapies; Pfizer; Sarepta
    Speaker or member of speakers bureau for: Alexion; argenx; Biogen; Catalyst; CSL Behring; Genentech; Grifols
    Research funding from: AMO Pharma; Astellas Gene Therapies; Biogen; Catalyst; Fulcrum; Novartis Gene Therapies; Pfizer; PTC Therapeutics; ReveraGen; Sarepta; Solid Biosciences 

Faculty

  • John F. Brandsema, MD

    Child Neurologist and Neuromuscular Section Head
    The Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania

     

    Disclosures

    John F. Brandsema, MD, has the following relevant financial relationships:
    Consultant or advisor for: AveXis/Novartis; Biogen; Genentech; Momenta/Janssen; NS Pharma; Pfizer; PTC Therapeutics; Sarepta; Scholar Rock
    Research funding from: Alexion; Astellas; AveXis/Novartis; Biogen; CSL Behring; FibroGen; Genentech; Pfizer; PTC Therapeutics; Sarepta
    Contracted researcher for: Alexion; Astellas; AveXis/Novartis; Biogen; CSL Behring; FibroGen; Genentech; Pfizer; PTC Therapeutics; Sarepta

  • Diana P. Castro, MD

    Neurologist With Special Qualification in Child Neurology
    Pediatric Neuromuscular Neurologist
    Medical Director, Neurology and Neuromuscular Care Center
    President, Neurology Rare Disease Center
    Dallas, Texas

     

    Disclosures

    Diana P. Castro, MD, has the following relevant financial relationships:
    Consultant or advisor for: Biogen; Novartis; PTC Therapeutics; Sarepta
    Research funding from: Biogen; FibroGen; Reveragen; Sarepta; Scholar Rock

  • Emma Ciafaloni, MD

    Professor of Neurology and Pediatrics
    University of Rochester
    Rochester, New York

     

    Disclosures

    Emma Ciafaloni, MD, has the following relevant financial relationships:
    Consultant or advisor for: Alexion; Amicus; argenx; Biogen; Dyne; Janssen; Momenta; NS Pharma; PTC Therapeutics; Sanofi; Sarepta Therapeutics; Scholar Rock; Viela Bio; Wave
    Research funding from: Sarepta Therapeutics

  • Basil T. Darras, MD

    Professor of Neurology
    Harvard Medical School
    Associate Neurologist-in-Chief
    Chief, Division of Clinical Neurology
    Director, Neuromuscular Center and SMA Program
    Boston Children's Hospital
    Boston, Massachusetts

     

    Disclosures

    Basil T. Darras, MD, has the following relevant financial relationships:
    Consultant or advisor for: Amicus, Inc.; Audentes; AveXis/Novartis Gene Therapies; Biogen; Pfizer; Roche/Genentech; Vertex
    Research funding from: AveXis; Biogen; FibroGen; Ionis Pharmaceuticals, Inc.; Novartis; PTC Therapeutics; Roche; Sarepta Pharmaceuticals; Scholar Rock
    Contracted researcher for: AveXis; Biogen; FibroGen; Ionis Pharmaceuticals, Inc.; Novartis; PTC Therapeutics; Roche; Sarepta Pharmaceuticals; Scholar Rock

  • Bakri H. Elsheikh, MBBS

    Professor of Neurology
    Division of Neuromuscular
    The Ohio State University
    Wexner Medical Center
    Columbus, Ohio

     

    Disclosures

    Bakri H. Elsheikh, MBBS, has the following relevant financial relationships:
    Consultant or advisor for: argenx; Biogen; Genentech
    Research funding from: argenx; Biogen; Celgene; Genentech; NMD Pharma; Pharnext; UCB

Editors

  • Meg Monday

    Senior Director, Content Development, Medscape, LLC
     

    Disclosures

    Meg Monday has no relevant financial relationships.

  • Pakinam Aboulsaoud, PharmD

    Senior Medical Education Director, Medscape, LLC

    Disclosures

    Pakinam Aboulsaoud, PharmD, has no relevant financial relationships.

Compliance Reviewer/Nurse Planner

  • Stephanie Corder, ND, RN, CHCP

    Associate Director, Accreditation and Compliance, Medscape, LLC

    Disclosures

    Stephanie Corder, ND, RN, CHCP, has no relevant financial relationships.

Peer Reviewer

This activity has been peer reviewed and the reviewer has no relevant financial relationships.


Accreditation Statements



In support of improving patient care, Medscape, LLC is jointly accredited with commendation 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.

    For Physicians

  • Medscape, LLC designates this enduring material for a maximum of 2.25 AMA PRA Category 1 Credit(s)™ . Physicians should claim only the credit commensurate with the extent of their participation in the activity.

    Contact This Provider

    For Nurses

  • Awarded 2.25 contact hour(s) of nursing continuing professional development for RNs and APNs; 1.0 contact hours are in the area of pharmacology.

    Contact This Provider

For questions regarding the content of this activity, contact the accredited provider for this CME/CE activity noted above. For technical assistance, contact [email protected]


Instructions for Participation and Credit

There are no fees for participating in or receiving credit for this online educational activity. For information on applicability and acceptance of continuing education credit for this activity, please consult your professional licensing board.

This activity is designed to be completed within the time designated on the title page; physicians should claim only those credits that reflect the time actually spent in the activity. To successfully earn credit, participants must complete the activity online during the valid credit period that is noted on the title page. To receive AMA PRA Category 1 Credit™, you must receive a minimum score of 75% on the post-test.

Follow these steps to earn CME/CE credit*:

  1. Read the target audience, learning objectives, and author disclosures.
  2. Study the educational content online or printed out.
  3. Online, choose the best answer to each test question. To receive a certificate, you must receive a passing score as designated at the top of the test. We encourage you to complete the Activity Evaluation to provide feedback for future programming.

You may now view or print the certificate from your CME/CE Tracker. You may print the certificate but you cannot alter it. Credits will be tallied in your CME/CE Tracker and archived for 6 years; at any point within this time period you can print out the tally as well as the certificates from the CME/CE Tracker.

*The credit that you receive is based on your user profile.

CME / CE

A Review of Rare Conditions Across the Lifespan: Pediatric Neuromuscular Disorders

Authors: Claudia A. Chiriboga, MD, MPH, FAAN; Perry B. Shieh, MD, PhD; John F. Brandsema, MD; Diana P. Castro, MD; Emma Ciafaloni, MD; Basil T. Darras, MD; Bakri H. Elsheikh, MBBSFaculty and Disclosures

CME / CE Released: 10/11/2022

Valid for credit through: 10/11/2023

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Contents of This CME Activity

All sections of this activity are required for credit.

Genetics, Epidemiology, and Gene Testing for Limb Girdle Muscular Dystrophy

Dr Brandsema discusses the pathophysiologic basics of LGMD.
John F. Brandsema, MD

Current and Emerging Approaches to Treatment of Limb Girdle Muscular Dystrophy: Is a Disease-Modifying Therapy on the Horizon?

Dr Brandsema discusses the current and emerging management approaches of patients with LGMD.
John F. Brandsema, MD

Strategies to Expedite Diagnosis in Duchenne Muscular Dystrophy: From Symptoms to Gene Testing

Dr Castro discusses strategies to reduce the time to diagnose in DMD.
Diana P. Castro, MD

Current and Emerging Therapies in Duchenne Muscular Dystrophy

Dr Ciafaloni discusses the current and emerging management approaches of DMD.
Emma Ciafaloni, MD

Opportunities and Challenges With Newborn Screening for Spinal Muscular Atrophy

Dr Chiriboga discusses strategies to reduce the time to diagnose in SMA.
Claudia A. Chiriboga, MD, MPH, FAAN

Data Round-Up of Disease-Modifying Therapies for Spinal Muscular Atrophy

Dr Darras discusses current management approaches of patients with SMA.
Basil T. Darras, MD

Best Practices for Adult Care in Spinal Muscular Atrophy

Dr Elsheikh discusses the management of adult patients with SMA.
Bakri H. Elsheikh, MBBS
 

Educational Impact Challenge

The goal of this activity is for learners to be better able to manage patients with pediatric neuromuscular disorders.

Before you begin this activity, please assess your clinical knowledge by completing this brief survey. Answering these questions again after the activity will allow you to see what you learned and to compare your answers with those of your peers.