Examples of Diseases that Utilize Gene‐Based Therapy

CME: 1.0

Target Audience

This activity is intended for primary care providers, internal-medicine specialists, pediatricians, pulmonologists, cardiologists, endocrinologists, gastroenterologists, hepatologists, neurologists, neuromuscular specialists, infectious-disease specialists, obstetricians/gynecologists, rheumatologists, dermatologists, hematologists, oncologists, neonatologists, genetic counselors, social workers, psychologists, nurse practitioners, physician assistants, nurses, and pharmacists

Learning Objectives

Upon completion of this activity, participants should be better able to:

  • Cite evidence for the use of gene‐based therapies that are approved or pending approval for the management of medical disorders
  • Counsel patients about clinical‐trial opportunities


Alessandra Biffi, MD

Professor of Pediatrics
Director, Division of Pediatric Hematology, 
Oncology and Stem Cell Transplant
University of Padua

Kevin Flanigan, MD

Director, Center for Gene Therapy 
Robert F. & Edgar T. Wolfe Foundation 
Endowed Chair in Neuromuscular Research 
Director, Nationwide Children’s Hospital 
Center for Research Translation in Muscular Dystrophy 
Therapeutic Development
Abigail Wexner Research Institute Nationwide Children’s Hospital
Professor of Pediatrics and Neurology
The Ohio State University

Kimberly Goodspeed, MD

Assistant Professor of Pediatrics, 
Neurology Division
University of Texas Southwestern Medical 
Center Dallas

Stephan A. Grupp, MD, PhD

Section Chief, Cellular Therapy and Transplant
Director, Cancer Immunotherapy Program
Children’s Hospital of Philadelphia
Novotny Professor of Pediatrics
Perelman School of Medicine at the 
University of Pennsylvania

John Kessler, MD

Davee Professor 
Davee Department of Neurology 
Northwestern University

Julie A. Parsons, MD

Haberfeld Family Endowed Chair in 
Pediatric Neuromuscular Disorders
Professor of Clinical Pediatrics and Neurology
University of Colorado School of Medicine
Children's Hospital Colorado

Perry B. Shieh, MD, PhD, FAAN

Professor of Neurology and Pediatrics
David Geffen School of Medicine at UCLA
University of California, Los Angeles

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