Listar por tema "Niemann-Pick Types A, B, /B"
Mostrando ítems 1-1 de 1
-
Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy
(2020) [Artículo de periódico]Background: Acid sphingomyelinase deficiency (ASMD) also known as Niemann-Pick disease, is a rare lysosomal storage disorder with a diverse disease spectrum that includes slowly progressive, chronic visceral (type B) and ...