Topic
Gene Targeted Therapies in Neuromuscular Diseases: Progress and Challenges

Presented By
Sabrina W. Yum, MD, associate professor of clinical neurology and pediatrics, Perelman School of Medicine at the University of Pennsylvania; medical director, CMTA Center of Excellence, Children’s Hospital of Philadelphia

Learning Objectives

• State genetic diagnostic and therapeutic advances in the field of neuromuscular diseases
• Review disease modifying therapies (gene therapy and RNA based therapies) for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), highlight challenges of gene therapy
• Discuss recent progress in Charcot-Marie-Tooth diseases

Target Audience
Physicians, nurse practitioners, nursing personnel, fellows, residents, medical students and other health care professionals who take care of children

Accreditation Statement
Tower Health is accredited by the Pennsylvania Medical Society to provide continuing medical education for physicians.

Tower Health designates this live activity for a maximum of 1 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Claiming CME Credits
Attendees MUST sign in with the six-digit activity code provided at the session using eeds within 24 hours of the conference start time and complete the online evaluation within 30 days at