Natural history of Fabry disease in females in the Fabry Outcome Survey von Deegan, P B, Baehner, A F, Barba Romero, M-Á, Hughes, D A, Kampmann, C, Beck, M

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Lysosomal delivery of therapeutic enzymes in cell models of Fabry disease von Marchesan, D., Cox, T. M., Deegan, P. B.

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Depression in adults with Fabry disease: A common and under-diagnosed problem von Cole, A. L, Lee, P. J, Hughes, D. A, Deegan, P. B, Waldek, S, Lachmann, R. H

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Monitoring enzyme replacement therapy in Fabry disease—Role of urine globotriaosylceramide von Whitfield, P. D., Calvin, J., Hogg, S., O'Driscoll, E., Halsall, D., Burling, K., Maguire, G., Wright, N., Cox, T. M., Meikle, P. J., Deegan, P. B.

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In-depth phenotyping for clinical stratification of Gaucher disease von D'Amore, Simona, Page, Kathleen, Donald, Aimée, Taiyari, Khadijeh, Tom, Brian, Deegan, Patrick, Tan, Chong Y, Poole, Kenneth, Jones, Simon A, Mehta, Atul, Hughes, Derralynn, Sharma, Reena, Lachmann, Robin H, Chakrapani, Anupam, Geberhiwot, Tarekegn, Santra, Saikat, Banka, Siddarth, Cox, Timothy M

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A randomised, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of three dosing schedules of agalsidase alfa enzyme replacement therapy for Fabry... von Hughes, D.A., Deegan, P.B., Milligan, A., Wright, N., Butler, L.H., Jacobs, A., Mehta, A.B.

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Clinical evaluation of biomarkers in Gaucher disease von Deegan, P B, Cox, T M

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Cardiac manifestations of fabry disease in female patients: a single centre experience von Peter, C U, Zhao, T X, Deegan, P B, Rusk, R A

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Prognostic value of troponin T in hemodialysis patients is independent of comorbidity von Deegan, Patrick B., Lafferty, Maureen E., Blumsohn, Aubrey, Henderson, Iain S., Mcgregor, Ellon

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Imiglucerase in the treatment of Gaucher disease: a history and perspective von Deegan, Patrick B, Cox, Timothy M

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Effects of switching from a reduced dose imiglucerase to velaglucerase in type 1 Gaucher disease: clinical and biochemical outcomes von van Dussen, Laura, Cox, Timothy M, Hendriks, Erik J, Morris, Elizabeth, Akkerman, Erik M, Maas, Mario, Groener, Johanna E M, Aerts, Johannes M F G, Deegan, Patrick B, Hollak, Carla E M

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Clinical evaluation of chemokine and enzymatic biomarkers of Gaucher disease von Deegan, Patrick B., Moran, Mary Teresa, McFarlane, Ian, Schofield, J. Paul, Boot, Rolf G., Aerts, Johannes M.F.G., Cox, Timothy M.

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Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document von Biegstraaten, Marieke, Arngrímsson, R, Barbey, F, Boks, L, Cecchi, Franco, Deegan, PB, Feldt-Rasmussen, Ulla, Geberhiwot, T, Germain, DP, Hendriksz, C, Hughes, DA, Kantola, I, Karabul, N, Lavery, C, Linthorst, GE, Mehta, A, van de Mheen, E, Oliveira, JP, Parini, R, Ramaswami, Uma, Rudnicki, M, Serra, A, Sommer, Claudia, Sunder-Plassmann, G, Svarstad, Einar, Sweeb, A, Terryn, W, Tylki-Szymanska, A, Tøndel, Camilla, Vujkovac, B, Weidemann, Frank, Wijburg, FA, Woolfson, P, Hollak, CE

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In-depth phenotyping for clinical stratification of Gaucher disease von D’Amore, Simona, Page, Kathleen, Donald, Aimée, Taiyari, Khadijeh, Tom, Brian, Deegan, Patrick, Tan, Chong Y, Poole, Kenneth, Jones, Simon A, Mehta, Atul, Hughes, Derralynn, Sharma, Reena, Lachmann, Robin H, Chakrapani, Anupam, Geberhiwot, Tarekegn, Santra, Saikat, Banka, Siddarth, Cox, Timothy M, Cox, T. M, Platt, F. M, Banka, S, Chakrapani, A, Deegan, P. B, Geberhiwot, T, Hughes, D. A, Jones, S, Lachmann, R. H, Santra, S, Sharma, R, Vellodi, A

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Clinical evaluation of biomarkers in Gaucher disease von Deegan, PB, Cox, TM

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Management of pain in Fabry disease in the UK clinical setting: consensus findings from an expert Delphi panel von Stepien, KM, Broomfield, A, Cole, D, Deegan, PB, Forshaw-Hulme, S, Hughes, D, Jovanovic, A, Morris, L, Muir, A, Ramaswami, U

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Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified D... von Hughes, DA, Aguiar, P, Deegan, PB, Ezgu, F, Frustaci, A, Lidove, O, Linhart, A, Lubanda, J-C, Moon, JC, Nicholls, K, Niu, D-M, Nowak, A, Ramaswami, U, Reisin, R, Rozenfeld, P, Schiffmann, R, Svarstad, E, Thomas, M, Torra, R, Vujkovac, B, Warnock, DG, West, ML, Johnson, J, Rolfe, MJ, Feriozzi, S

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Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document von Biegstraaten, Marieke, Arngrímsson, R, Barbey, F, Boks, L, Cecchi, Franco, Deegan, PB, Feldt-Rasmussen, Ulla, Geberhiwot, T, Germain, DP, Hendriksz, C, Hughes, DA, Kantola, I, Karabul, N, Lavery, C, Linthorst, GE, Mehta, A, van de Mheen, E, Oliveira, JP, Parini, R, Ramaswami, Uma, Rudnicki, M, Serra, A, Sommer, Claudia, Sunder-Plassmann, G, Svarstad, Einar, Sweeb, A, Terryn, W, Tylki-Szymanska, A, Tøndel, Camilla, Vujkovac, B, Weidemann, Frank, Wijburg, FA, Woolfson, P, Hollak, CE

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Energy Conversion Alternatives Study (ECAS), Westinghouse phase 1. Volume 7: Metal vapor Rankine topping-steam bottoming cycles von Deegan, P. B.

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Transfer of high cost drugs to NICE risks fragmentation of care of rare diseases von Hendriksz, Chris J

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