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Year Number of Results
2001 1
2005 4
2006 2
2007 1
2010 1
2011 5
2012 8
2013 2
2014 2
2015 2
2017 1
2019 1
2020 2
2021 2
2022 1
2023 2
2024 1

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32 results

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Page 1
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.
Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes DA, Hopkin RJ, Tøndel C, Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A, Deegan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B, Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Sakov A, Warnock DG. Wallace EL, et al. Among authors: deegan pb. J Med Genet. 2024 May 21;61(6):520-530. doi: 10.1136/jmg-2023-109445. J Med Genet. 2024. PMID: 37940383 Free PMC article. Clinical Trial.
Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study.
Deegan PB, Goker-Alpan O, Geberhiwot T, Hopkin RJ, Lukina E, Tylki-Szymanska A, Zaher A, Sensinger C, Gaemers SJM, Modur V, Thurberg BL, Sharma J, Najafian B, Mauer M, DasMahapatra P, Wilcox WR, Germain DP. Deegan PB, et al. Mol Genet Metab. 2023 Feb;138(2):106963. doi: 10.1016/j.ymgme.2022.11.002. Epub 2022 Nov 9. Mol Genet Metab. 2023. PMID: 36481125 Free PMC article. Clinical Trial.
Clinical evaluation of biomarkers in Gaucher disease.
Deegan PB, Cox TM. Deegan PB, et al. Acta Paediatr Suppl. 2005 Mar;94(447):47-50; discussion 37-8. doi: 10.1111/j.1651-2227.2005.tb02111.x. Acta Paediatr Suppl. 2005. PMID: 15895712 Review.
Fabry disease in females: clinical characteristics and effects of enzyme replacement therapy.
Deegan PB, Bähner F, Barba M, Hughes DA, Beck M. Deegan PB, et al. In: Mehta A, Beck M, Sunder-Plassmann G, editors. Fabry Disease: Perspectives from 5 Years of FOS. Oxford: Oxford PharmaGenesis; 2006. Chapter 30. In: Mehta A, Beck M, Sunder-Plassmann G, editors. Fabry Disease: Perspectives from 5 Years of FOS. Oxford: Oxford PharmaGenesis; 2006. Chapter 30. PMID: 21290688 Free Books & Documents. Review.
Clinical Features of Lysosomal Acid Lipase Deficiency.
Burton BK, Deegan PB, Enns GM, Guardamagna O, Horslen S, Hovingh GK, Lobritto SJ, Malinova V, McLin VA, Raiman J, Di Rocco M, Santra S, Sharma R, Sykut-Cegielska J, Whitley CB, Eckert S, Valayannopoulos V, Quinn AG. Burton BK, et al. Among authors: deegan pb. J Pediatr Gastroenterol Nutr. 2015 Dec;61(6):619-25. doi: 10.1097/MPG.0000000000000935. J Pediatr Gastroenterol Nutr. 2015. PMID: 26252914 Free PMC article.
Mechanistic convergence and shared therapeutic targets in Niemann-Pick disease.
Colaco A, Kaya E, Adriaenssens E, Davis LC, Zampieri S, Fernández-Suárez ME, Tan CY, Deegan PB, Porter FD, Galione A, Bembi B, Dardis A, Platt FM. Colaco A, et al. Among authors: deegan pb. J Inherit Metab Dis. 2020 May;43(3):574-585. doi: 10.1002/jimd.12191. Epub 2019 Dec 5. J Inherit Metab Dis. 2020. PMID: 31707734 Free PMC article.
Atypical presentation of late-onset Sandhoff disease: a case report.
Salamon A, Szpisjak L, Zádori D, Lénárt I, Maróti Z, Kalmár T, Brierley CMH, Deegan PB, Klivényi P. Salamon A, et al. Among authors: deegan pb. Ideggyogy Sz. 2021 Nov 30;74(11-12):425-429. doi: 10.18071/isz.74.0425. Ideggyogy Sz. 2021. PMID: 34856081 Free article. English.
32 results