Potter, Jane E. http://orcid.org/0000-0002-8109-9983
Petts, Gemma
Ghosh, Arunabha
White, Fiona J.
Kinsella, Jane L.
Hughes, Stephen
Roberts, Jane
Hodgkinson, Adam
Brammeier, Kathryn
Church, Heather
Merrigan, Christine
Hughes, Joanne
Evans, Pamela
Campbell, Helen
Bonney, Denise
Newman, William G.
Bigger, Brian W.
Broomfield, Alexander
Jones, Simon A.
Wynn, Robert F.
Article History
Received: 27 January 2021
Accepted: 4 May 2021
First Online: 21 May 2021
Declarations
:
: No specific ethical approval for this study was required. All samples and treatments/interventions were collected and given as part of standard clinical monitoring, treatment and management of Wolman disease patients. Use and analysis of histology was carried out under REC08H101063.
: The multimodal patients’ parental/legal guardians signed written consent for this publication and use/analysis of histological images consent was taken using REC08H101063. The exception is patient 1 who had passed away, specific consent for use of genetics and histology images was not acquired and they have therefore not been included.
: Authors had no competing interests unless listed below. SJ—has received consultancy fees, grants, and travel support for conferences from Alexion Pharmaceuticals, Inc. AG—has received consultancy fees/honoraria from Alexion Pharmaceuticals, Inc. FW—has received consultancy fees/honoraria from Alexion Pharmaceuticals, Inc.