Niemann-Pick Disease Type B

Watchlist

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Retrieved

2021-01-23

Source

Orphanet

Trials

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Genes

SMPD1, ABCA1, CHIT1, ICAM1, IGF1, CCL18, GULP1, NPB, H19

Drugs

Acetylleucine, Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus, Recombinant human acid sphingomyelinase

Acetylleucine, Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus, Recombinant human acid sphingomyelinase, Ursodeoxycholic acid ( DELURSAN, URSO, URSOLVAN ), haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate

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Niemann-Pick disease type B is a mild subtype of Niemann-Pick disease, an autosomal recessive lysosomal disease, and is characterized clinically by onset in childhood with hepatosplenomegaly, growth retardation, and lung disorders such as infections and dyspnea