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Pediatric Neuromuscular Disorders
This course explores both diagnosis and treatment of neuromuscular diseases in children. Pulmonary function testing and ventilation strategies will be covered in detail. Anyone who cares for children with neuromuscular disease will benefit from these presentations. A final presentation will cover integrative palliative care to aid in the care of patients and families who are experiencing neuromuscular disease.
Video presentations included:
- Making the Diagnosis: Diagnostic Strategies in Neuromuscular Disease (Oren Kupfer, MD)
- Pulmonary Function Testing for Monitoring and Management (Sandeep Puranik, MBBS, DCH, MD)
- New and Evolving Therapeutics in Neuromuscular Disease Management (Mai Elmallah, MD, MS)
- Ventilation Strategies for Acute and Long-Term Management of Neuromuscular Disease (Moshe Prero, MD)
- Palliative Care in Neuromuscular Disorders: An Approach to Guiding Patients and Families Through Care Decisions (Lauren Treat, MD)
This product is part of Pediatric Core series presented at the 2022 ATS International Conference.
Target Audience
Pediatric pulmonary and critical care physicians who work in a clinical setting and are currently engaged in maintenance of certification.
Learning Objectives
Making the Diagnosis: Diagnostic Strategies in Neuromuscular Disease (Oren Kupfer, MD)
At the end of this activity, participants will be able to describe the clinical features and diagnostic tests for:
- Infantile-onset neuromuscular disorders (0-1 years old).
- Early childhood-onset neuromuscular disorders (1-10 years old).
- Late childhood-onset neuromuscular disorders (11-20 years old).
Pulmonary Function Testing for Monitoring and Management (Sandeep Puranik, MBBS, DCH, MD)
- Describe the Pulmonary Function Tests (PFTs) used in patients with Neuromuscular Disorders.
- Utilize PFT data to assess and monitor the level of respiratory morbidity and disease trajectory. Optimize patient management based on PFT data.
- Recognize the limitations of using PFTs in weak neuromuscular patients.
New and Evolving Therapeutics in Neuromuscular Disease Management (Mai Elmallah, MD, MS)
- Briefly describe the mechanisms that result in respiratory impairment in SMA and DMD
- Compare and Contrast gene replacement therapy and gene editing
- Identify FDA approved and investigational gene therapy for SMA and DMD and describe how these therapies attenuate the disease.
- Recognize the limitations of gene therapy for SMA and DMD and describe the ongoing trials that are attempting to address these barriers.
Ventilation Strategies for Acute and Long-Term Management of Neuromuscular Disease (Moshe Prero, MD)
- Describe the indications for initiating ventilation in the patient with neuromuscular weakness- Delineate the different modes of ventilation available
- Differentiate between strategies for short term and long term ventilatory support
- Recall the recommended setting and circumstances in which ventilator support can be initiated
- Identify strategies to “troubleshoot” common ventilatory problems and scenarios.
Palliative Care in Neuromuscular Disorders: An Approach to Guiding Patients and Families Through Care Decisions (Lauren Treat, MD)
- Describe the ethical framework for shared decision making with attention to the clinician perspective on treatments that are impermissible, permissible, or obligatory.
- Compare and contrast common decision points in neuromuscular disease with considerations for both the outpatient and inpatient setting.
- Distinguish between the principles of pediatric palliative care as they relate to shared decision making, highlighting the role of primary and specialty palliative care involvement.
| Name | Title | Institution | Role | Relationship |
| Başak Çoruh, MD | Associate Professor | University of Washington | Core Curriculum Chair | No relationships to disclose |
| Margaret (Molly) M. Hayes, MD, ATSF | Assistant Professor of Medicine | Beth Israel Deaconess Medical Center | Core Curriculum Chair | No relationships to disclose |
| Jane Gross, MD | Assistant Professor | National Jewish Health | Pediatric Core Chair | Cystic Fibrosis Foundation: Research - investigator initiated American Board of Pediatrics, Pulmonology Subboard: Consultant |
| Carolyn Okorie, MD | Clinical Assistant Professor | Stanford University | Pediatric Core Co-Chair | No relationships to disclose |
| Michael McCown, DO | Pediatric Pulmonologist | Walter Reed National Military Medical Center | Pediatric Core Co-Chair | No relationships to disclose |
| Carmen Leon Astudillo, MD | Clinical Assistant Professor | University of Florida Health | Planner | No relationships to disclose |
| Fei Jamie Dy, MD | Assistant Professor | UMass Chan Medical School | Planner | No relationships to disclose |
| Oren Kupfer, MD | Associate Professor of Pediatrics | Children's Hospital Colorado | Faculty presenter | No relationships to disclose |
| Sandeep Puranik, MBBS, DCH, MD | Assistant Professor of Clinical Pediatrics | Riley Hospital for Children | Faculty presenter | No relationships to disclose |
| Mai Elmallah, MD, MS | Associate Professor of Pediatrics | Duke University Medical School | Faculty presenter | No relationships to disclose |
| Moshe Prero, MD | Assistant Professor of Pediatrics | Rainbow Babies and Children's Hospital | Faculty presenter | No relationships to disclose |
| Lauren Treat, MD | Child Neurologist, Palliative Medicine Physician | Children’s Hospital Colorado | Faculty presenter | No relationships to disclose |
Accreditation Statement
The American Thoracic Society is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
Disclosure Declaration
It is the policy of the ATS to ensure independence, balance, objectivity, scientific rigor, and integrity in all of its continuing education activities. As an accredited CME provider, the ATS requires that its planners, reviewers and presenters comply with the ACCME Standards for Integrity and Independence in Accredited Continuing Education. The ATS also requires specific disclosure of relationships with companies and organizations associated with tobacco or cannabis and prohibits or limits participation of faculty in official ATS activities, including CME, if any tobacco industry relationship or some types of cannabis industry relationships are present. To see the most recent policies regarding potential conflicts of interest as well as the mechanisms to resolve such conflicts, press the COI Policy link below.
This educational activity may include discussion of unapproved uses of a drug, product, or device. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.
Core Curriculum Chairs
Başak Çoruh, MD
Margaret (Molly) M. Hayes, MD, ATSF
Pediatric Core Chairs
Jane Gross, MD
Carolyn Okorie, MD
Michael McCown, DO
Planners
Carmen Leon Astudillo, MD
Fei Jamie Dy, MD
Faculty
Oren Kupfer, MD
Sandeep Puranik, MBBS, DCH, MD
Mai Elmallah, MD, MS
Moshe Prero, MD
Lauren Treat, MD
Instructions to Receive Credit
To earn credit for this course, follow these instructions:
- Launch and view all session videos.
- After viewing the videos, you must attest that you viewed all the videos prior to starting the post test.
- You must pass the post test with a 70% score or better.
- Complete the Course Evaluation.
- Once you view the videos, pass the post test and complete the evaluation, you may claim credits.
- To view your transcript and print your certificate, go to the My Account drop-down list. Then select Transcript.
Available Credit
- 3.00 ABP MOCSuccessful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 3.00 MOC points in the American Board of Pediatrics’ (ABP) Maintenance of Certification (MOC) program. Participants will earn MOC.
- 3.00 AMA PRA Category 1 Credit(s)™The American Thoracic Society designates this for a maximum of 3.00 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
- 3.00 Participation