Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency.
about
Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase DeficiencyDisease manifestations and burden of illness in patients with acid sphingomyelinase deficiency (ASMD)From promising molecules to orphan drugs: Early clinical drug developmentLysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.Consensus recommendation for a diagnostic guideline for acid sphingomyelinase deficiency.Human acid sphingomyelinase structures provide insight to molecular basis of Niemann-Pick disease.Sphingomyelin-induced inhibition of the plasma membrane calcium ATPase causes neurodegeneration in type A Niemann-Pick disease.Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months.Enhanced Delivery and Effects of Acid Sphingomyelinase by ICAM-1-Targeted Nanocarriers in Type B Niemann-Pick Disease Mice.Types A and B Niemann-Pick disease.
P2860
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P2860
Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency.
description
2015 nî lūn-bûn
@nan
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
2015年论文
@zh
2015年论文
@zh-cn
name
Successful within-patient dose ...... d sphingomyelinase deficiency.
@ast
Successful within-patient dose ...... d sphingomyelinase deficiency.
@en
type
label
Successful within-patient dose ...... d sphingomyelinase deficiency.
@ast
Successful within-patient dose ...... d sphingomyelinase deficiency.
@en
prefLabel
Successful within-patient dose ...... d sphingomyelinase deficiency.
@ast
Successful within-patient dose ...... d sphingomyelinase deficiency.
@en
P2093
P2860
P1476
Successful within-patient dose ...... d sphingomyelinase deficiency.
@en
P2093
Ana Cristina Puga
Beth L Thurberg
Elias Shamiyeh
George A Diaz
Gerald F Cox
Haig Inguilizian
Handrean Soran
Kerry Culm-Merdek
Melissa P Wasserstein
Natalie Lippa
P2860
P356
10.1016/J.YMGME.2015.05.013
P577
2015-05-30T00:00:00Z