Understanding patient and parent/caregiver perceptions on gene therapy in Gaucher disease: an international survey

por Collin-Histed, Tanya, Rosenberg, Aviva, Hopman, Noortje, Pacey, Jessica
Publicado 2023

International consensus on clinical severity scale use in evaluating Niemann–Pick disease Type C in paediatric and adult patients: results from a Delphi Study

por Evans, William, Patterson, Marc, Platt, Frances, Guldberg, Christina, Mathieson, Toni, Pacey, Jessica
Publicado 2021