2024 PPMD DUCHENNE HEALTHCARE PROFESSIONALS SUMMIT

January 17, 2024 to January 19, 2024

PPMD hosts an annual Duchenne Healthcare Professionals Summit to convene clinicians, researchers, industry partners, and representatives from government health agencies to discuss the current field of Duchenne and Becker muscular dystrophy. PPMD coordinates a robust agenda to discuss relevant topics, disseminate new data and information, and encourage collaboration to discuss potential innovative approaches to treatment.

Target Audience

  • Allied Health Professionals
  • Nurses
  • Nurse Practitioners
  • Physicians
  • Physician Assistants

Learning Objectives

  • Identifying barriers in researching treatments and advancing care of patients living with Duchenne muscular dystrophy.
  • Reporting current state of newborn screening for dystrophinopathies (CK testing) including legislation and implementation on both state and federal levels.
  • Examining barriers to Spanish-speaking individuals participating in clinical trials and research studies for patients with Duchenne muscular dystrophy.
  • Assessing the perceived ethical dilemmas around clinical trials conducted in rare disease patient populations.
  • Summarizing the evolution of research and learnings about the roles and functions of dystrophin proteins since the discovery of the DMD gene in 1986.
  • Presenting a family genetic case study, diagnostic process, and strategies to measure muscle function in patients with Becker muscular dystrophy.
  • Examining a theoretical case in which a patient with Duchenne experiences endocrine-related issues including weight gain, delayed pubertal development, and impaired bone density.
  • Reviewing the importance of advocacy at the federal level in rare disease and the impact it has on research investment, investigating and approving new drugs, and access to therapies.
  • Describing the impact of the patients and family experience during a recent FDA advisory committee, which led to a successful FDA review and subsequent approval of a novel new therapy for patients with Duchenne muscular dystrophy.
  • Reviewing the research, learnings, care advancements, and innovation in the field of Duchenne muscular dystrophy in the past 40 years as experienced by one leading physician who helped pioneer the field.
  • Examining potentials for specialized screening, novel approaches to anxiety management, and what psychiatric medications may be prescribed for patients with Duchenne muscular dystrophy who exhibit behavioral issues.
  • Discussing the small cohort of dystrophinopathy patients who do not fit into a clear Duchenne or Becker diagnosis, and how they should be recognized, cared for, and investigated further to better understand their disease trajectory and care approaches.
  • Discussing unknown safety profiles, access and reimbursement barriers, timing of initiation and other important aspects of potential combination therapies for patients with Duchenne muscular dystrophy.
  • Reviewing the various challenges the FDA faces when reviewing new therapies in rare disease communities.
  • Explaining emerging genetic technology and new generations of gene therapy that are in the early stages of research that could potentially treat patients with Duchenne muscular dystrophy in the future.
  • Discussing priorities and expectations of families affected by the diagnosis of Duchenne and the providers who care for these patients.
  • Reviewing  the various types of outcome measures currently used in clinical trials for Duchenne muscular dystrophy and each of their their limitations and barriers.
  • Predicting the potential advances, forward-thinking ideas, and innovative technology applied in the field of Duchenne muscular dystrophy.
Course summary
Available credit: 
  • 15.50 AMA PRA Category 1 Credit(s)™
  • 15.50 ANCC
  • 15.50 Attendance
  • 1.55 IACET CEU
  • 15.50 JA Credit - AH
Registration Opens: 
01/19/2024
Registration Expires: 
01/18/2025
Activity Starts: 
01/17/2024 - 9:00am EST
Activity Ends: 
01/19/2024 - 1:00pm EST
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Durham, NC 27705
United States

Kim Phung, MD

Joel Wood

Buddy Cassidy

Peter Marks

Niki Armstrong, MS, CGC

Francesco Muntoni

Norah Crossnohere, PhD

Art Caplan, PhD

Leigh Ramos-Platt, MD

Katherine Beaverson

Ash Dugar, PhD, MBA

Amanda Wilkison, RN, BSN

Molly Colvin, PhD

Aravind Veerapandiyan, MD

Natalie Truba, PhD

Elizabeth Margolis, DO

Claudia Senesac, PT, PhD

Jerry Mendell, MD

 

  • AMA PRA Category 1 Credit(s)
  • ANCC
  • Attendance
  • IACET CEU
  • JA Credit - AH

Available Credit

  • 15.50 AMA PRA Category 1 Credit(s)™
  • 15.50 ANCC
  • 15.50 Attendance
  • 1.55 IACET CEU
  • 15.50 JA Credit - AH
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