- PALYNZIQ® (pegvaliase-pqpz) Injection for PKU (BMRN)
- HMI-102 Gene Therapy (FIXX)
- RTX-134 (RUBY)
A Deep Dive Into Phenylketonuria (PKU) - Exploring New Approaches by BioMarin, Homology, Rubius, and ModernaTicker(s): BMRN, MRNA, RUBY, FIXX
Name: Dr Barbara Burton - MD
- 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.
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