| Title : Twice weekly dosing with Sebelipase alfa (Kanuma) rescues severely ill infants with Wolman disease - de Castro_2024_Orphanet.J.Rare.Dis_19_244 |
| Author(s) :
de Castro MJ , Jones SA , de Las Heras J , Sanchez-Pintos P , Couce ML , Colon C , Crujeiras P , Unceta M , Church H , Brammeier K , Yee WH , Cooper J , Lopez de Frutos L , Serrano-Gonzalo I , Camba MJ , White FJ , Holmes V , Ghosh A |
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Ref :
Orphanet J Rare Dis , 19 :244 , 2024 |
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Abstract :
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| PubMedSearch : de Castro_2024_Orphanet.J.Rare.Dis_19_244 |
| PubMedID: 38918870 |
| Gene_locus related to this paper:
human-LIPA |