The impacts of olipudase alfa on adults with ASMD: The patient-reported experience
Donnelly, Conan B., Mbua, Solomon, Crowe, Joslyn, Hopkin, Justin
Published in Molecular genetics and metabolism (01.02.2024)
Published in Molecular genetics and metabolism (01.02.2024)
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Journal Article
Development of a patient-reported data collection system for Niemann-Pick disease
Donnelly, Conan B., Imrie, Jackie, Mbua, Solomon, Bolton, Shaun
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
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Journal Article
Acid sphingomyelinase deficiency: Burden of disease and real world impact of enzyme replacement therapy on pediatric patients and caregivers
Hopkin, Justin, Donnelly, Conan, Crowe, Joslyn, Mathieson, Toni, Mbua, Solomon
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
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Journal Article