Module 1 - Current Evidence for Gene Replacement Therapies in Duchenne Muscular Dystrophy

Primary Audience:

This activity is intended to meet the educational needs of clinicians involved in the care of patients with Duchenne muscular dystrophy

This module focuses on the understanding of recent advancements in gene therapy, provide a clear interpretation of the most current clinical trial results, and consider their implications for patient care.

Abdullah AlQahtani, MD, MPH

Department of Neurology
Neuromuscular Medicine
Johns Hopkins Sidney Kimmel Comprehensive Cancer Center
Baltimore, MD

Abdullah AlQahtani specializes in inherited and noninherited neuromuscular neurological diseases. He treats patients who have amyotrophic lateral sclerosis (ALS) and spinal and bulbar muscular atrophy (also known as Kennedy disease). He also performs electromyography and neuromuscular ultrasound. 

Dr. AlQahtani obtained his medical degree from the King Saud University in Saudi Arabia. He also completed an internship and earned a master’s degree in public health from the University of Miami Miller School of Medicine, and he obtained residency training in neurology at the University Hospitals of Cleveland-Case Western Reserve University and the Louis Stokes Cleveland Department of Veterans Affairs Medical Center. After completing a neuromuscular fellowship at Case Western Reserve University, Dr. AlQahtani joined the neurogenetics branch of the National Institute of Neurological Disorders and Stroke.

His research focuses on identification of biomarkers of disease progression in patients with spinal and bulbar muscular atrophy.

Sabrina Yum, MD

Associate Professor of Clinical Neurology
Clinical Director, CMTA Center of Excellence, Children’s Hospital of Philadelphia
Department of Neurology
Children’s Hospital of Pennsylvania
Philadelphia, PA

Dr. Sabrina Wai Yum, MD is a clinical neurophysiologist in Philadelphia, PA and has over 40 years of experience in the medical field. She graduated from Indiana University School of Medicine in 1983. She is affiliated with medical facilities such as Milton S Hershey Medical Center and Hospital of the University of Pennsylvania.

1.
Integrate new data on novel and emerging treatments into DMD treatment algorithms.