Dual Therapy and the Changing Treatment Landscape of SMA
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Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.
In this Mind Moments episode, Aaron Zelikovich, MD, a neuromuscular physician at Lenox Hill Hospital, part of Northwell Health, joins the podcast to discuss the emerging conversation around dual therapy in spinal muscular atrophy (SMA). As disease-modifying treatments continue to reshape survival and long-term outcomes, Zelikovich examines whether combining SMN-targeted approaches could provide additional benefit, while emphasizing that evidence supporting dual therapy remains limited and significant barriers to studying it persist.
The conversation explores clinician and patient interest in combination treatment, insurance and access challenges, and what current evidence suggests about adding therapy after gene replacement, particularly in children who are not meeting expected motor milestones. Zelikovich also discusses the need for better biomarkers to guide treatment decisions, the changing natural history of adult SMA, and why stronger real-world data and prospective research will be critical to determining whether dual therapy ultimately has a place in SMA care.
Looking for more Neuromuscular discussion? Check out the NeurologyLive® Neuromuscular clinical focus page.
Episode Breakdown:
- 1:05 – Clinical perspectives and current controversy surrounding dual therapy in SMA
- 2:40 – Discussing dual therapy expectations and uncertainty with patients and families
- 4:35 – Evidence guiding when clinicians should consider adding a second therapy
- 6:20 – Neurology News Minute
- 8:55– Developing better biomarkers to guide long-term SMA treatment decisions
- 11:15 – Research needed to evaluate dual therapy and adult SMA outcomes
- 13:35 – Moving the dual therapy conversation forward through patient-clinician partnerships
The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here:
FDA Clears Roche's Single-Biomarker Elecsys pTau217 Blood Test for Alzheimer Disease
FDA Extends Deramiocel Review for Duchenne Muscular Dystrophy After HOPE-3 Amendment
AHA/ASA Issue New Guideline for Adult Stroke Rehabilitation and Recovery
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