Expert Interview
Discussing the potential of enzyme replacement therapies such as Sanofi's Nexviazyme (avalglucosidase alfa-ngpt) and Amicus Therapeutics' Pombiliti + Opfolda (AT-GAA) in late-onset Pompe disease with a Genetic Counselor.
Ticker(s): FOLD, SNYInstitution: Lurie Children’s Hospital of Chicago
- Genetic Counselor
- treats approximately 50 LOPD patients (the majority are children/adolescents)
- familiar with Amicus Therapeutics's Pombiliti + Opfolda(AT-GAA) and the Phase 3 Propel Study
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