In vivo enrichment of transduced cells to enhance gene therapy for Fabry disease von Nagree, Murtaza S., Faber, Mary L., Tate, Everett, Schroeder, Mark A., DiPersio, John F., Medin, Jeffrey A.

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Hemoglobinopathies von Atweh, George F, DeSimone, Joseph, Saunthararajah, Yogen, Fathallah, Hassana, Weinberg, Rona S, Nagel, Ronald L, Fabry, Mary E, Adams, Robert J

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Properties of the mouse α‐globin HS‐26: Relationship to HS‐40, the major enhancer of human α‐globin gene expression von Bouhassira, Eric E., Kielman, Menno F., Gilman, John, Fabry, Mary F., Suzuka, Sandy, Leone, Ofelia, Gikas, Ekatarinas, Bernini, Luigi F., Nagel, Ronald L.

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Measuring patient experiences in Fabry disease: validation of the Fabry-specific Pediatric Health and Pain Questionnaire (FPHPQ) von Ramaswami, Uma, Stull, Donald E, Parini, Rossella, Pintos-Morell, Guillem, Whybra, Catharina, Kalkum, Gisela, Rohrbach, Marianne, Raluy-Callado, Mireia, Beck, Michael, Chen, Wen-Hung, Wiklund, Ingela

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The myocardial phenotype of Fabry disease pre-hypertrophy and pre-detectable storage von Augusto, João B, Johner, Nicolas, Shah, Dipen, Nordin, Sabrina, Knott, Kristopher D, Rosmini, Stefania, Lau, Clement, Alfarih, Mashael, Hughes, Rebecca, Seraphim, Andreas, Vijapurapu, Ravi, Bhuva, Anish, Lin, Linda, Ojrzyńska, Natalia, Geberhiwot, Tarekegn, Captur, Gabriella, Ramaswami, Uma, Steeds, Richard P, Kozor, Rebecca, Hughes, Derralynn, Moon, James C, Namdar, Mehdi

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Enzyme replacement therapy improves peripheral nerve and sweat function in Fabry disease von Schiffmann, Raphael, Floeter, Mary Kay, Dambrosia, James M., Gupta, Surya, Moore, David F., Sharabi, Yehonatan, Khurana, Ramesh K., Brady, Roscoe O.

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Functional characterisation of alpha-galactosidase a mutations as a basis for a new classification system in fabry disease von Lukas, Jan, Giese, Anne-Katrin, Markoff, Arseni, Grittner, Ulrike, Kolodny, Ed, Mascher, Hermann, Lackner, Karl J, Meyer, Wolfgang, Wree, Phillip, Saviouk, Viatcheslav, Rolfs, Arndt

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Antibody Epitope of Human α‐Galactosidase A Revealed by Affinity Mass Spectrometry: A Basis for Reversing Immunoreactivity in Enzyme Replacement Therapy of Fabry Disease von Kukacka, Zdenek, Iurascu, Marius, Lupu, Loredana, Rusche, Hendrik, Murphy, Mary, Altamore, Lorenzo, Borri, Fabio, Maeser, Stefan, Papini, Anna Maria, Hennermann, Julia, Przybylski, Michael

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A detailed pathologic examination of heart tissue from three older patients with Anderson–Fabry disease on enzyme replacement therapy von Sheppard, Mary N, Cane, Paul, Florio, Richard, Kavantzas, Nicholas, Close, Lydia, Shah, Jaymin, Lee, Philip, Elliott, Perry

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Blocking hyperactive androgen receptor signaling ameliorates cardiac and renal hypertrophy in Fabry mice von Shen, Jin-Song, Meng, Xing-Li, Wight-Carter, Mary, Day, Taniqua S, Goetsch, Sean C, Forni, Sabrina, Schneider, Jay W, Liu, Zhi-Ping, Schiffmann, Raphael

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The heart in Fabry's disease von Sheppard, Mary N

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A New Era for Rare Genetic Diseases: Messenger RNA Therapy von Martini, Paolo G V, Guey, Lin T

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α-Galactosidase A Expressed in the Salivary Glands Partially Corrects Organ Biochemical Deficits in the Fabry Mouse Through Endocrine Trafficking von PASSINEAU, Michael J, FAHRENHOLZ, Timothy, KINGSTON, H. M, BENZA, Raymond L, MACHEN, Laurie, ZOURELIAS, Lee, NEGA, Katherine, PAUL, Rachel, MACDOUGALL, Mary J, MAMAEVA, Olga, STEET, Richard, BARNES, Jarrod

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Cardiovascular magnetic resonance demonstration of the spectrum of morphological phenotypes and patterns of myocardial scarring in Anderson-Fabry disease von Deva, Djeven Parameshvara, Hanneman, Kate, Li, Qin, Ng, Ming Yen, Wasim, Syed, Morel, Chantal, Iwanochko, Robert M, Thavendiranathan, Paaladinesh, Crean, Andrew Michael

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First phenotypic description of a female patient with c.610 T > C variant of GLA: a renal-predominant presentation of Fabry disease von Greillier, Sophie, Daniel, Laurent, Caillaud, Catherine, Dussol, Bertrand, Touchard, Guy, Goujon, Jean-Michel, Jourde-Chiche, Noémie, Bobot, Mickaël

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Wideband Low-Profile Fabry-Perot Cavity Antenna with Metasurface von Song, Xueyan, Dong, Ang, Li, XuPing, Zhang, YunQi, Lin, Haoyuan, Yang, Hailong, Li, Yapeng

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Management of pain in Fabry disease in the UK clinical setting: consensus findings from an expert Delphi panel von Stepien, Karolina M, Broomfield, Alexander, Cole, Duncan, Deegan, Patrick B, Forshaw-Hulme, Stuart, Hughes, Derralynn, Jovanovic, Ana, Morris, Liz, Muir, Alison, Ramaswami, Uma

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Prevalence of CADASIL and Fabry Disease in a Cohort of MRI Defined Younger Onset Lacunar Stroke von Kilarski, Laura L, Rutten-Jacobs, Loes C A, Bevan, Steve, Baker, Rob, Hassan, Ahamad, Hughes, Derralynn A, Markus, Hugh S

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Out-of-plane modes in three-dimensional Fabry-Perot microlasers von Sobeshchuk, N., Guidry, M. A., Lafargue, C., Gashemi, R., Decanini, D., Zyss, J., Lebental, M.

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Psychological aspects of patients with Fabry disease von Crosbie, T. Wilson, Packman, Wendy, Packman, S.

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