The use of Ambroxol for the treatment of Gaucher disease: A systematic review by Diego Agustín Abelleyra Lastoria, Simranjeet Grewal, Derralynn Hughes

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Inflammation in Fabry disease: stages, molecular pathways, and therapeutic implications by Hibba Kurdi, Hibba Kurdi, Lucia Lavalle, Lucia Lavalle, James C. C. Moon, James C. C. Moon, Derralynn Hughes, Derralynn Hughes

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Estimation of Health State Utility Values in Fabry Disease Using Vignette Development and Valuation by Derralynn Hughes, Andrew Lenny, Koonal Shah, Louise Longworth, Giovanna Devercelli, Olulade Ayodele

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An integrated discrete event simulation and particle swarm optimisation model for optimising efficiency of cancer diagnosis pathways by Elizabeth A. Cooke, Nadia A.S. Smith, Spencer A. Thomas, Carolyn Ruston, Sukhraj Hothi, Derralynn Hughes

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Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events by Michael Beck, Derralynn Hughes, Christoph Kampmann, Guillem Pintos-Morell, Uma Ramaswami, Michael L. West, Roberto Giugliani

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Biomarkers of Myocardial Fibrosis: Revealing the Natural History of Fibrogenesis in Fabry Disease Cardiomyopathy by Patrício Aguiar, Olga Azevedo, Rui Pinto, Jacira Marino, Carlos Cardoso, Nuno Sousa, Damião Cunha, Derralynn Hughes, José Luís Ducla Soares

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Shining a light on an additional clinical burden: work-related digital communication survey study – COVID-19 impact on NHS staff wellbeing by Ameet Bakhai, Leah McCauley, Liba Stones, Saria Khalil, Jay Mehta, Nicola Price, Vinodh Krishnamurthy, Lucy H. H. Parker, Derralynn Hughes

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Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: A Fabry Outcome Survey analysis by Michael Beck, Derralynn Hughes, Christoph Kampmann, Sylvain Larroque, Atul Mehta, Guillem Pintos-Morell, Uma Ramaswami, Michael West, Anna Wijatyk, Roberto Giugliani

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Changes in Angiogenesis and Bone Turnover Markers in Patients with Gaucher Disease Developing Osteonecrosis by Simona D’Amore, Kenneth Eric Poole, Uma Ramaswami, Derralynn Hughes, Kathleen Page, Antonio Giovanni Solimando, Angelo Vacca, Timothy Martin Cox, Patrick Deegan

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Long-term follow-up of renal function in patients treated with migalastat for Fabry disease by Daniel G. Bichet, Roser Torra, Eric Wallace, Derralynn Hughes, Roberto Giugliani, Nina Skuban, Eva Krusinska, Ulla Feldt-Rasmussen, Raphael Schiffmann, Kathy Nicholls

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Corrigendum to “Long-term follow-up of renal function in patients treated with migalastat for Fabry disease” [Bichet et al., MGM Reports; 28 (2021) 100786] by Daniel G. Bichet, Roser Torra, Eric Wallace, Derralynn Hughes, Roberto Giugliani, Nina Skuban, Eva Krusinska, Ulla Feldt-Rasmussen, Raphael Schiffmann, Kathy Nicholls

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Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom by Lena Hubig, Anna-Katrine Sussex, Alasdair MacCulloch, Derralynn Hughes, Ryan Graham, Liz Morris, Syed Raza, Andrew J. Lloyd, Amanda Sowinski, Katy Gallop

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

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Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in... by Maureen Cleary, James Davison, Rachel Gould, Tarekegn Geberhiwot, Derralynn Hughes, Jean Mercer, Alexandra Morrison, Elaine Murphy, Saikat Santra, James Jarrett, Swati Mukherjee, Karolina M. Stepien

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Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study by Aleš Linhart, Gabriela Dostálová, Kathy Nicholls, Michael L. West, Camilla Tøndel, Ana Jovanovic, Pilar Giraldo, Bojan Vujkovac, Tarekegn Geberhiwot, Einat Brill-Almon, Sari Alon, Raul Chertkoff, Rossana Rocco, Derralynn Hughes

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Transition of patients with Gaucher disease type 1 from pediatric to adult care: results from two international surveys of patients and health care professionals by Karolina M. Stepien, Irena Žnidar, Beata Kieć-Wilk, Beata Kieć-Wilk, Angel Jones, Daniela Castillo-García, Magy Abdelwahab, Shoshana Revel-Vilk, Shoshana Revel-Vilk, Ella Lineham, Derralynn Hughes, Uma Ramaswami, Tanya Collin-Histed

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A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease by Barry J. Byrne, Tarekegn Geberhiwot, Bruce A. Barshop, Richard Barohn, Derralynn Hughes, Drago Bratkovic, Claude Desnuelle, Pascal Laforet, Eugen Mengel, Mark Roberts, Peter Haroldsen, Kristin Reilley, Kala Jayaram, Ke Yang, Liron Walsh, on behalf of the POM-001/002 Investigators

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P006: Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in Fabry disease: 2-Year interim results from BRIGHT51 by John Bernat, Myrl Holida, Nicola Longo, Ozlem Goker-Alpan, Eric Wallace, Patrick Deegan, Camilla Tondel, Francois Eyskens, Ulla Feldt-Rasmussen, Derralynn Hughes, Antonio Pisani, Ales Linhart, Rossana Rocco, Einat Almon, Sari Alon, Raul Chertkoff, David Warnock, Stephen Waldek, William Wilcox

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A 15-Year Perspective of the Fabry Outcome Survey by Roberto Giugliani MD, PhD, Dau-Ming Niu MD, PhD, Uma Ramaswami MD, Michael West MD, Derralynn Hughes MD, Christoph Kampmann MD, PhD, Guillem Pintos-Morell MD, PhD, Kathleen Nicholls MD, Jörn-Magnus Schenk MD, PhD, Michael Beck MD

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

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