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Expert Interview

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

Reviewing the long-Term Data from BridgeBio's Phase 2 Study of Glycosylated Alpha-dystroglycan (⍺DG) levels as a Surrogate Endpoint in Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)

Ticker(s): BBIO

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 Goal
This call will focus on the long-term data from its Phase 2 trial in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) at the Annual Congress of World Muscle Society (WMS) providing support for the possible use of glycosylated ⍺DG levels as a surrogate endpoint in the ongoing Phase 3 study for accelerated approval.

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Reason

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