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| Title : Outcome of haematopoietic cell transplantation in children with lysosomal acid lipase deficiency: a study on behalf of the EBMT Inborn Errors Working Party - |
| Author(s) : Lum SH , Minkov M , Jones SA , Hazelaar S , Sirait T , Potter JE , Stepensky P , Garban F , Pichler H , Stein J , Kaya Z , Schulz A , Mellgren K , Diaz de Heredia C , Pochon C , Riesco S , Diaz MA , Michel G , Lindemans C , Gruhn B , Albert MH , Lankester AC , Neven B , Wynn R |
| Ref : Bone Marrow Transplant , : , 2023 |
| PubMedID: 36788366 |
| Gene_locus related to this paper: human-LIPA |
| Title : Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease - Potter_2021_Orphanet.J.Rare.Dis_16_235 |
| Author(s) : Potter JE , Petts G , Ghosh A , White FJ , Kinsella JL , Hughes S , Roberts J , Hodgkinson A , Brammeier K , Church H , Merrigan C , Hughes J , Evans P , Campbell H , Bonney D , Newman WG , Bigger BW , Broomfield A , Jones SA , Wynn RF |
| Ref : Orphanet J Rare Dis , 16 :235 , 2021 |
| Abstract : Potter_2021_Orphanet.J.Rare.Dis_16_235 |
| ESTHER : Potter_2021_Orphanet.J.Rare.Dis_16_235 |
| PubMedSearch : Potter_2021_Orphanet.J.Rare.Dis_16_235 |
| PubMedID: 34020687 |
| Gene_locus related to this paper: human-LIPA |