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A Deep Dive Into Phenylketonuria (PKU) - Exploring New Approaches by BioMarin, Homology, Rubius, and Moderna


Who's the expert?

Name: Dr Barbara Burton - MD

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 Goal
To dig into the PKU treatment landscape and examine where the competing therapies from  BioMarin, Homology, Rubius, and Moderna stand. This call will discuss:
  • PALYNZIQ® (pegvaliase-pqpz) Injection for PKU (BMRN)
  • HMI-102 Gene Therapy (FIXX)
  • RTX-134 (RUBY)
  • mRNA-3283(MRNA)

Are You Interested In These Questions?

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