| 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 |
| Gene_locus related to this paper: human-LIPA |
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 (2021)
Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease
Orphanet J Rare Dis
16 :235
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 (2021)
Orphanet J Rare Dis
16 :235