Applying Clinical Data to the Management of Patients with Spinal Muscular Atrophy

Applying Clinical Data to the Management of Patients with Spinal Muscular Atrophy

Program Overview

This continuing education activity includes five, 30-minute modules that will relay information to learners regarding the treatment of patients with spinal muscular atrophy (SMA). Dr. Basil T. Darras will interview three experts in the field of SMA, covering topics including a scientific overview, the standardized scales for motor function, and how to manage patients with SMA. Dr. Robert J. Graham will then serve as the interviewer and Dr. Darras, the interviewee, where they will discuss the characteristics and diagnostic criteria of spinal muscular atrophy, along with emerging clinical trial data. Each module is worth 0.5 CME/CNE credits. Each module will provide healthcare professionals further insight on spinal muscular atrophy and how to apply it into their clinical practice.

Target Audience

Neurologists, pediatricians, primary care physicians, internists, nurse practitioners, nurses, and other healthcare professionals involved in the management of patients with SMA.

Learning Objectives

  1. Understand the current science and diagnostic criteria for SMA
  2. Describe the mechanism of action of newly approved and emerging therapies to treat patients with SMA
  3. Analyze the safety and efficacy data for new and emerging therapies for the treatment of patients with SMA
  4. Incorporate shared decision making and evidence-based data to the management of patients with SMA


  1. Scientific Overview: The Pathology, Biology, and Molecular Genetics of SMA
  2. Understanding the Characteristics of SMA and Diagnostic Criteria
  3. Common Standardized Scales for Measuring Motor Function in SMA
  4. Clinical Trial Data: New and Emerging Therapies for SMA
  5. Management of Patients with SMA: Standards of Care


Basil T. Darras, MD
Associate Neurologist-in-Chief
Chief, Division of Clinical Neurology
Director, Neuromuscular Center and Spinal Muscular Atrophy Program
Boston Children’s Hospital
Professor of Neurology
Harvard Medical School
Boston, MA


Robert J. Graham, MD
Director, C.A.P.E. and Home Ventilation Program
Associate in the Division of Critical Care Medicine
Department of Anesthesiology, Perioperative and Pain Medicine
Boston Children’s Hospital
Boston, MA


John F. Brandsema, MD
Attending Physician
Neuromuscular/General Neurology & Electromyography
Neuromuscular Education Director and
Neuromuscular Section Head
Children’s Hospital of Philadelphia
Philadelphia, PA


Amy Pasternak, PT, DPT, PCS
Boston University
Boston Children’s Hospital
Boston, MA

Financial Support

This activity has been supported by an independent educational grant from Biogen.

Provider Information

Provided by