Clinical Insights on Recent Advances in Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a leading, fatal, autosomal recessive disorder that affects approximately one in 10,000 live births. Nusinersen was approved as the first targeted therapy for SMA, and since its groundbreaking approval data has been published that demonstrates its efficacy for both infantile- and later-onset SMA populations. Additionally, recent guidelines were published that provide evidence-based recommendations for the diagnosis and management of SMA.

However, despite these advances there are knowledge gaps among health care providers (HCPs) regarding current data on safety and efficacy of approved and emerging therapies, which can result in treatment delays. Closing these gaps will promote early and accurate diagnosis and optimal treatment of patients with SMA.

In this CME Outfitters live and on demand webcast, expert faculty will host a case-based panel discussion that focuses on screening and early diagnosis of SMA and the importance of utilizing the latest clinical data on the efficacy and safety of approved and emerging therapies.

Learning Objectives

At the end of this CE activity, participants should be able to:

- Review the pathophysiology and therapeutic targets of SMA.
- Apply the current recommendations for screening and early diagnosis of SMA.
- Evaluate the latest clinical data on the efficacy and safety of approved and emerging therapies for SMA.

The following learning objectives pertain only to those requesting CNE or CPE credit:

- Review the pathophysiology and therapeutic targets of SMA.
- Describe the current recommendations for screening and early diagnosis of SMA.
- Evaluate the latest clinical data on the efficacy and safety of approved and emerging therapies for SMA.