Spinal Muscular Atrophy: A New Era of Evaluation and Treatment
Section: Academy of Pediatric PT
Session Code: PD-2B-6414
Date: Friday, January 25, 2019
Time: 11:00 AM - 1:00 PM
Location: Marriott Marquis
Room: Marquis Salon 5
Session Type: Educational Sessions
Session Level: Intermediate
Spinal muscular atrophy (SMA) is an autosomal recessive disorder leading to progressive muscle weakness across a spectrum, from infantile to adult onset. In 2016, Spinraza (nusinersen) became the first drug approved for the treatment SMA. Similarly, a promising gene therapy resulted in individuals with SMA type 1 achieving motor milestones outside of historical expectations. This session will present the recent groundbreaking progress made in the treatment of SMA and review the evolving natural history of the disease, including research-based evidence and a unique clinical experience. The speakers will identify the pros and cons of each treatment, providing clinicians with information necessary to help guide a potential family.Attendees will learn about the current outcome measures used for the assessment of individuals with SMA, suggested modifications based on clinical experience, and a rationale that emphasizes this need for change. The speakers will present the new natural history쳌 of SMA development seen in treated individuals, highlight the divergence from previous expectations, and identify patient characteristics that are emerging as efficacy modifiers. Patient videos will provide examples of new movement patterns to illustrate the difficulties with current assessments and support the development of new outcomes in this changing cohort.The presenters will explain considerations for ongoing care and therapeutic interventions, comparing recommendations for a traditional, untreated cohort and treated patient populations. Clinicians will gain a better understanding of potential gains to assist with goal setting, treatment planning, and dosing recommendations across treatment settings.
Learning Objectives: Upon completion of this course, you will be able to:
1. Describe the evolution of the spinal muscular atrophy (SMA) phenotype and compare and contrast the differences between historical presentation and the new natural history.쳌
2. Explain the results of recent clinical trials and the current drug development pipeline of other compounds.
3. Interpret the clinical and functional relevance of proposed modifications to traditionally utilized clinical outcome measures in SMA.
4. Develop and apply an understanding of the evaluation and treatment recommendations for untreated and treated individuals with SMA across treatment settings (eg, early intervention, school, outpatient, etc).
Search sessions by day, speaker, section, keyword, event type, or display all sessions.