US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

Medical Educational Resource

We're accelerating the delivery of treatments and cures.

Transitions in Care in SMA Patient #1: Transition planning following adolescent diagnosis

Activity Snapshot

Activity Type: 
Print/PDF
Release Date: 
Wednesday, April 13, 2022

Faculty

John Brandsema, MD
Neuromuscular Education Director and Neuromuscular Section Head
Children’s Hospital of Philadelphia Philadelphia, PA
Activity Overview: 

This case study will explore transitions in care in SMA in patient #1 (15-year-old-male). This case will focus on transition planning following adolescent diagnosis.

Learning Objectives: 
  • Identify factors that affect disease course in spinal muscular atrophy (SMA)
    • SMA subtype, time of onset or diagnosis, vigilant respiratory and multidisciplinary care, approved therapies, etc.
  • Understand the differing needs of pediatric vs adult patients with SMA, with emphasis on the importance of psychosocial support for adult patients
  • Implement proper medical management for transitions in care, according to best practice guidelines that help address issues specifically encountered by patients with SMA