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A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative.
Collin-Histed T, Stoodley M, Beusterien K, Elstein D, Jaffe DH, Revel-Vilk S, Davies EH; International Gaucher Alliance (IGA). Collin-Histed T, et al. Among authors: davies eh. Orphanet J Rare Dis. 2023 Jul 21;18(1):195. doi: 10.1186/s13023-023-02828-w. Orphanet J Rare Dis. 2023. PMID: 37480076 Free PMC article.
The definition of neuronopathic Gaucher disease.
Schiffmann R, Sevigny J, Rolfs A, Davies EH, Goker-Alpan O, Abdelwahab M, Vellodi A, Mengel E, Lukina E, Yoo HW, Collin-Histed T, Narita A, Dinur T, Revel-Vilk S, Arkadir D, Szer J, Wajnrajch M, Ramaswami U, Sidransky E, Donald A, Zimran A. Schiffmann R, et al. Among authors: davies eh. J Inherit Metab Dis. 2020 Sep;43(5):1056-1059. doi: 10.1002/jimd.12235. Epub 2020 Apr 3. J Inherit Metab Dis. 2020. PMID: 32242941 Free PMC article.
Protocol for a mixed-methods study to develop and feasibility test a digital system for the capture of patient-reported outcomes (PROs) in patients receiving chimeric antigen receptor T-cell (CAR-T) therapies (the PRO-CAR-T study).
Hughes SE, McMullan C, Aiyegbusi OL, Shaw K, Kinsella F, Ferguson P, Khatsuria F, Burns D, Pyatt L, Ansell J, Chakera E, Richardson-Abraham J, Denniston AK, Davies EH, Craddock C, Calvert M. Hughes SE, et al. Among authors: davies eh. BMJ Open. 2024 Mar 29;14(3):e085392. doi: 10.1136/bmjopen-2024-085392. BMJ Open. 2024. PMID: 38553074 Free PMC article.
Recommendations to address respondent burden associated with patient-reported outcome assessment.
Aiyegbusi OL, Cruz Rivera S, Roydhouse J, Kamudoni P, Alder Y, Anderson N, Baldwin RM, Bhatnagar V, Black J, Bottomley A, Brundage M, Cella D, Collis P, Davies EH, Denniston AK, Efficace F, Gardner A, Gnanasakthy A, Golub RM, Hughes SE, Jeyes F, Kern S, King-Kallimanis BL, Martin A, McMullan C, Mercieca-Bebber R, Monteiro J, Peipert JD, Quijano-Campos JC, Quinten C, Rantell KR, Regnault A, Sasseville M, Schougaard LMV, Sherafat-Kazemzadeh R, Snyder C, Stover AM, Verdi R, Wilson R, Calvert MJ. Aiyegbusi OL, et al. Among authors: davies eh. Nat Med. 2024 Mar;30(3):650-659. doi: 10.1038/s41591-024-02827-9. Epub 2024 Feb 29. Nat Med. 2024. PMID: 38424214 Review.
101 results