Expert Interview
A look at enzyme replacement therapies in Pompe disease including Sanofi's Nexviazyme and Amicus Therapeutics' Pombiliti + Opfolda (AT-GAA)
Ticker(s): FOLD, SNYInstitution: Cincinnati Children's Hospital Medical Center
- Professor of Clinical Pediatrics and Director of the Medical Genetics Residency and Fellowship Programam at Cincinnati Children's Hospital Medical Center.
- Currently manages 15 patients with late-onset pompe disease (LOPD) and has experience prescribing Nexviazyme.
- Research focuses on Fabry disease, Pompe disease, hypophosphatasia, velocardiofacial syndrome, 1p36 deletion syndrome, Neurofibromatosis type I, and other genetic conditions.
Please describe the impact of AT-GAA's expected switch-only label on your use of the therapy within Pompe.
Added By: user1a5m4kWhat portion of the currently treated Nexviazyme and Myozyme do you plan to switch to AT-GAA over the coming 1 mo, 3 mos, 1 yr, 3 yrs, etc?
Please describe your understanding of the change in treated Pompe prevalence over the past few years? What is driving this growth?
Added By: user1a5m4kAre You Interested In These Questions?
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