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Expert Interview
Slingshot members are talking to an expert! The topic is:
Discussing the PTC Therapeutics' Phase 3 APHENITY data on Sepiapterin in Phenylketonuria (PKU)
Ticker(s): PTCTWho's the expert?
Institution: Northwestern
- Professor of Pediatrics (Genetics, Birth Defects, and Metabolism) at Northwestern University Feinberg School of Medicine & Director, PKU Program.
- Currently manages 40 patients with Pompe disease and is familiar with the literature on alglucosidase alfa (AT2221). Also, manages 320 patients with Phenylketonuria (PKU).
- Participated in numerous clinical trials of new therapies for metabolic disorders including PKU, lysosomal storage disorders and fatty acid oxidation defects and has authored over 200 articles in the peer-reviewed literature.
Interview GoalThis conversation will focus on the standard of care and the potential of Sepiapterin (formerly PTC923) an oral formulation of synthetic sepiapterin for the treatment of phenylketonuria (PKU) including the results of the phase 3 APHENITY trial.
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