Derralynn Hughes

Evaluating the relationship between antidrug antibodies and efficacy and safety outcomes in patients with Fabry disease receiving enzyme replacement therapy: a systematic literature review

Patrício Aguiar, John A. Bernat, Derralynn Hughes

BACKGROUND: Fabry disease is a lysosomal storage disorder caused by deficient activity of the enzyme α-galactosidase A, resulting in progressive accumulation of globotriaosylceramide (Gb3) and widespread tissue and organ damage. Enzyme replacement therapy (ERT) can slow disease progression but may elicit antidrug …

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Additional file 1 of Evaluating the relationship between antidrug antibodies and efficacy and safety outcomes in patients with Fabry disease receiving enzyme replacement therapy: a systematic literature review

Patricio Aguiar, John A. Bernat, Derralynn Hughes

Supplementary Material 1

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Additional file 1 of Evaluating the relationship between antidrug antibodies and efficacy and safety outcomes in patients with Fabry disease receiving enzyme replacement therapy: a systematic literature review

Patricio Aguiar, John A. Bernat, Derralynn Hughes

Supplementary Material 1

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Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study

Myrl Holida, Aleš Linhart, Nicola Longo, Eric (Trombonist) Wallace et autres

BACKGROUND: Enzyme replacement therapies (ERTs) approved for Fabry disease require infusions every 2 weeks (E2W). Pegunigalsidase alfa, a PEGylated ERT with a prolonged half-life vs. other ERTs, may allow extension of the dosing interval to every 4 weeks (E4W). BRIGHT F51 (NCT03614234) …

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Cardiovascular Morbidity and Mortality in Fabry Disease

Emanuele Monda, Athanasios Bakalakos, Annamaria Del Franco, R Lillo et autres

BACKGROUND: Cardiac involvement is the main determinant of adverse outcomes in Fabry disease. The study aimed to investigate cardiovascular outcomes in patients with Fabry disease. METHODS: This was a multicenter, retrospective, longitudinal study of consecutively referred adult patients with Fabry disease. The …

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Lucerastat effect on kidney function in patients with Fabry disease: Results from the ongoing open label-extension (OLE) of the phase 3 clinical program

Eric Wallace, Dominique P. Germain, Peter Blattmann, Aline Frey et autres

Impact of mobility-aid use on late-onset Pompe disease (LOPD) patient experience: Insights from patient interviews

Derralynn Hughes, Skyler Jackson, Patti A. Engel, Austin R. Letcher et autres

Associations of physical activity and sedentary behaviour with mental health and quality of life in adults with Fabry disease

Sarah Gosling, Cherry Kilbride, Louise Mansfield, Derralynn Hughes et autres

Joint arthroplasty is an effective treatment option in managing hip pain in Gaucher disease

Krishan Almeida, Kaifeng Liang, Derralynn Hughes, Uma Ramaswami et autres

Co-producing UK clinical guidelines for lysosomal disorders: A patient professional partnership

Sophie Thomas, Toni Mathieson, Alexander Broomfield, Simon A. Jones et autres

Empowering sustainable Gaucher communities

Tanya L. Collin-Histed, Vesna Aleksovska, Christian J. Hendriksz, Derralynn Hughes et autres

Inflammation and lysosomal dysfunction in Fabry macrophages

Veronica Lentini, Giuseppe Uras, David Moreno Martínez, Derralynn Hughes