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ELIKIDS: Baseline characteristics from the eliglustat substrate reduction therapy trial in children with Gaucher disease type 1 or type 3
by
Giraldo, Pilar
,
Gokcay, Gulden
,
Batsu, Isabela
,
Kissell, Julie
,
Perichon, Maria G.
,
Drelichman, Guillermo I.
Published in
Molecular genetics and metabolism
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