touchMEETING HIGHLIGHTS Insights into evolving needs of patients with SMA as they age: claims data and expert opinion
Watch leading spinal muscular atrophy (SMA) experts discuss current trends in pharmacologic and non-pharmacologic therapy use, barriers to use of these standards of care, and potential solutions to improve access as patients age.
In this animation, the key results of a claims study assessing standard of care therapy use in SMA will be presented, including expert opinion on current barriers to utilization and potential solutions.
In these symposium highlights, three SMA experts discuss current utilization of pharmacologic and non-pharmacologic therapies in SMA, including current barriers to use and solutions, particularly during the transition from pediatric to adult care.
In this chapter, understand the learning objectives and meet the faculty members participating in the Cure SMA symposium.
In this chapter, Dr Randal Richardson highlights key US claims data on pharmacologic therapy use as patients age.
In this chapter, Dr Leslie Nelson highlights key US claims data on physical and occupational therapy utilization, barriers to non-pharmacologic therapy use and potential solutions to reduce those barriers.
In this chapter, Dr Mathula Thangarajh discusses the current use of speech therapy in SMA and improving its integration into multidisciplinary care.
In this chapter, the expert faculty discuss unmet needs in people transitioning from pediatric to adult care.
In this chapter, the expert panel provide their key takeaway messages for the care of people with SMA.
Overview & Learning Objectives
Overview
People with SMA have complex needs, requiring multidisciplinary support and the use of non-pharmacological therapies such as physical, occupational, and speech therapy, and more recently, pharmacological treatments.1–3 However, numerous barriers may limit access to these standard of care treatments, particularly as patients transition from pediatric to adult care.4 In these symposium highlights and animation, learn about the current utilization of standard of care therapy, barriers to access and potential solutions to improve care.
Learning Objectives
After watching this activity, participants should be better able to:
- Review a claims data study evaluating care patterns for people with SMA
- Use claims data study to discuss the existing barriers and challenges to care access in adolescents and adults with SMA
- Discuss ongoing efforts and future directions in eliminating disparities in care for all people with SMA
About Faculty
Dr Randal Richardson
Gillette Children’s Specialty Healthcare Saint Paul, MN, USA
Randal Richardson, MD, MMS is a pediatric neurologist at Gillette Children’s Specialty Healthcare specializing in the care of people with SMA. He obtained his medical degree from Brown University Alpert Medical School, Providence, Rhode Island and holds a Master’s of Medical Science in Neurophysiology.
Dr Richardson discloses: Advisory board fees from Biogen, Novartis, Genentech, Scholar Rock, NS Pharma, and Sarepta. Safety monitoring committee fees for Syneos Health. Institutional research funds from Biogen, Novartis, Genentech, Scholar Rock, and Avidity.
Dr Leslie Nelson
UT Southwestern Medical Center Dallas, TX, USA
Leslie Nelson, MPT, PhD is a physical therapist at the University of Texas Southwestern Medical Center in Dallas, Texas, where she also holds teaching responsibilities. She has been a specialist in neuromuscular disorders including SMA for two decades and is involved in training clinical evaluators for clinical trials and the development of clinically relevant measures for trials.
Dr Nelson discloses: Consultant and advisory board member for Biogen and Scholar Rock. Consultant for Roche and Novartis. Speaker for Genentech.
Dr Mathula Thangarajh
Virginia Commonwealth University Richmond, VA, USA
Mathula Thangarajh, MD, PhD is a pediatric neurologist and Neuromuscular Physician at the Virginia Commonwealth University
Richmond, Virgina. She is a fellow of the American Academy of Neurology.
Dr Thangarajh discloses: Speaker fees from NS Pharma. Grant funding from the MDA and NIH.
References
- Mercuri E, Finkel RS, Muntoni F, et al. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord. 2018;28(2):103-115.
- Crisafulli S, Boccanegra B, Vitturi G, et al. Pharmacological Therapies of Spinal Muscular Atrophy: A Narrative Review of Preclinical, Clinical–Experimental, and Real-World Evidence. Brain Sci. 2023;13(10):1446.
- McGrattan KE, Graham RJ, DiDonato CJ, et al. Dysphagia Phenotypes in Spinal Muscular Atrophy: The Past, Present, and Promise for the Future. Am J Speech Lang Pathol. 2021;30:1008-22.
- Walter MC, Chiriboga C, Duong T, et al. Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era. J Neuromuscul Dis. 2021;8(4):543-551.
