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Taking a Look at the Changing Landscape for Spinal Muscular Atrophy After AAN 2019 and Pending FDA Decision On Zolgensma's Approval

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Who's the expert?

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.

Interview Questions

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?

Added By: joe_mccann

Characterize advantages, disadvantages of Spinraza. How does real-world use compare to clinical trial data?

Added By: joe_mccann

Is Zolgensma a cure? Should the “cure” word be used?

Added By: joe_mccann

Can you, should you, compare Zolgensma to Spinraza? How do the respective therapies compare across efficacy, safety, convenience?

Added By: joe_mccann

Will SMA patients on Spinraza today switch to Zolgensma? Can you walk through that decision-making process?

Added By: joe_mccann

What percentage of your patients do you think might be treated with Zolgensma over the next year? Five years?

Added By: joe_mccann

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?

Added By: joe_mccann

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