Activity ID



August 30, 2019

Format Type


CME Credit



CME Provider

The France Foundation


This activity, offered as a live grand rounds and an online enduring activity, focuses on gene replacement therapy, using spinal muscular atrophy (SMA) as an example of its potential impact on patients. This education aims to inform the entire care team and improve knowledge about gene therapy, SMA and the importance of an early diagnosis, and how gene therapy is impacting patients with SMA. This activity, developed by a multidisciplinary team of experts, will answer questions about what gene therapy is and how it works.

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NOTE: If a Member Board has not deemed this activity for MOC approval as an accredited CME activity, this activity may count toward an ABMS Member Board’s general CME requirement. Please refer directly to your Member Board’s MOC Part II Lifelong Learning and Self-Assessment Program Requirements.

Educational Objectives

• Explain how gene therapy works
• Review SMA genetics and diagnosis
• Explore potential implications of gene replacement therapy for SMA
• Summarize key clinical trial data on emerging treatments for SMA, with a focus on gene replacement therapy


Gene replacement therapy, Spinal muscular atrophy, neuromuscular, pediatric neurology, neurology


Medical Knowledge, Patient Care & Procedural Skills, Practice-based Learning & Improvement, Systems-based Practice

CME Credit Type

AMA PRA Category 1 Credit

Practice Setting

Academic Medicine, Inpatient, Outpatient, Physician Scientists, Rural, Urban

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