Taking a Look at the Changing Landscape for Spinal Muscular Atrophy After AAN 2019 and Pending FDA Decision On Zolgensma's ApprovalTicker(s): NVS, BIIB, RHHBY
Name: Dr Edward Smith - MD
Institution: Duke University
- Co-Director of Duke Children's Neuromuscular Program & Professor of Neurology and Pediatrics at Duke.
- Treats hundreds of neuromuscular patients, including over 60 Spinal Muscular Atrophy patients and 80 Duchenne MD patients (all ages).
- Has administered over 250 doses of Spinraza since April 2017.
- Actively involved in clinical research and trials with a focus on neuromuscular diseases; PI on multiple DMD studies including an Exon 53 skipping specific study.
Questions to come from Adam Feuerstein and Damian Garde of STAT News!Added By: joe_mccann
Three years ago, there were no medicines to treat SMA. Today, there is one (Spinraza) approved, a second (Zolgensma) on the cusp of approval, and a third (risdiplam) not far behind. What has this meant for the SMA community? Where do you see the disease and its therapies evolving from here?
Characterize advantages, disadvantages of Spinraza. How does real-world use compare to clinical trial data?
Is Zolgensma a cure? Should the “cure” word be used?
Can you, should you, compare Zolgensma to Spinraza? How do the respective therapies compare across efficacy, safety, convenience?
Will SMA patients on Spinraza today switch to Zolgensma? Can you walk through that decision-making process?
What percentage of your patients do you think might be treated with Zolgensma over the next year? Five years?
There has been a lot of discussion about the (proposed) high cost of Zolgensma. It's likely to be the most expensive medicine in the world. How do you view the cost benefit of a gene therapy for SMA?
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