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Expert Interview

Slingshot members are talking to an expert! The topic is:

A Second View: Delving into the MARINA Phase 1/2 clinical trial data on Avidity Biosciences' AOC 1001 in treating Myotonic Dystrophy

Ticker(s): RNA

Who's the expert?

Institution: University of Washington

  • Associate Professor of Neurology at University of Washington and a researcher at the Seattle Wellstone Center.
  • Manages ~15 patients with myotonic dystrophy type 1 (DM1).
  • Researches focuses on inclusion body myositis, facioscapulohumeral muscular dystrophy, and ALS (funded throug the NIH), and clinical interests focus on neuromuscular disorders, such as motor neuron disease (ALS), myasthenia gravis, peripheral neuropathies and muscular dystrophies including FSHD.

Interview Questions
Q1.

What is the current standard of care for patients with Myotonic Dystrophy Type 1?

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Q2.

On a scale of 1 to 10, how excited are you about AOC 1001 based on this most recent data?

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Q3.

Are there other treatments currently in development for Myotonic Dystrophy which excite you and how do they compare to AOC 1001?

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Reason

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