Expanding Potential for Long-Term Treatment in SMA
Episode 95, Aug 11, 2023, 10:00 AM
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 episode, Thomas Crawford, MD, a pediatric neurologist at Johns Hopkins Medicine, spoke on the recently published 5-year analysis of the NURTURE study (NCT02386553), a long-term trial assessing the efficacy and safety of nusinersen (Spinraza; Biogen) in presymptomatic infants with spinal muscular atrophy (SMA). Crawford discussed the significance of the positive findings, the shift in conversations around treatment optimization in SMA, and how subgroup data may factor into the design of future trials.
Looking for more Neuromuscular Disorders discussion? Check out the NeurologyLive® neuromuscular clinical focus page.
Episode Breakdown:
In this episode, Thomas Crawford, MD, a pediatric neurologist at Johns Hopkins Medicine, spoke on the recently published 5-year analysis of the NURTURE study (NCT02386553), a long-term trial assessing the efficacy and safety of nusinersen (Spinraza; Biogen) in presymptomatic infants with spinal muscular atrophy (SMA). Crawford discussed the significance of the positive findings, the shift in conversations around treatment optimization in SMA, and how subgroup data may factor into the design of future trials.
Looking for more Neuromuscular Disorders discussion? Check out the NeurologyLive® neuromuscular clinical focus page.
Episode Breakdown:
- 1:20 – Benefits seen with nusinersen in NURTURE
- 4:10 – Changes in goals for treating SMA
- 6:05 – Complexities with getting infants therapy days after diagnosis
- 9:40 – Neurology News Minute
- 12:30 – Subgroup findings from NURTURE
- 15:20 – Ways to improve treatment optimization in SMA
This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.
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:
- World Health Organization Adds Several MS Treatments to List of Essential Medicines
- Ceribell's Status Epilepticus Software Receives FDA Clearance With CMS NTAP Coverage Included
- FDA Accepts New Drug Application for Long-Acting Form of Glatiramer Acetate
- Essential Tremor Agent Ulixacaltamide Continues to Show Positive Results in Essential1 Study
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