Expert Interview
Discussing investigational therapies for the treatment of Hunter Syndrome, including DNL310 and RGX-121
Ticker(s): DNLI, RGNXInstitution: 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.
What do you think of the data shown so far on DNL310?
Added By: wilson_adminWhat do you think of the data shown so far on RGX-121?
Added By: wilson_adminDo you anticipate the need for an ERT following gene therapy treatment? If so, at what point should it be added? Which ERT would be best suited following gene therapy?
Added By: jsharon3What are the considerations for deciding between DNL310 and RGX121 in the treatment of neuronopathic patients?
Added By: jsharon3What role do you anticipate DNL310 playing in the treatment of attenuated patients? What type and age range of attenuated patients will be best suited for treatment with DNL310?
Added By: jsharon3Are You Interested In These Questions?
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