Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment.
about
Therapeutic potential of intracerebroventricular replacement of modified human β-hexosaminidase B for GM2 gangliosidosisLess Is More: Substrate Reduction Therapy for Lysosomal Storage DisordersRethinking fatigue in Gaucher diseasePsychiatric manifestations of treatable hereditary metabolic disorders in adultsMiglustat Reverts the Impairment of Synaptic Plasticity in a Mouse Model of NPC DiseaseIminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff diseaseGastrointestinal disturbances and their management in miglustat-treated patients.Lyso-GM2 ganglioside: a possible biomarker of Tay-Sachs disease and Sandhoff diseaseDevelopment of targeted therapies for Parkinson's disease and related synucleinopathiesTherapeutic approaches for lysosomal storage diseasesAtypical presentation of late-onset Tay-Sachs diseaseNewborn screening for lysosomal storage disorders.Emerging therapies for neurodegenerative lysosomal storage disorders - from concept to reality.Lysosomal storage diseases--the horizon expands.Treatment of lysosomal storage disorders: successes and challenges.Infantile gangliosidoses: Mapping a timeline of clinical changes.Substrate reduction therapy for Krabbe's disease.Small molecules as therapeutic agents for inborn errors of metabolism.Protein misfolding diseases: prospects of pharmacological treatment.Effect of cyclic, low dose pyrimethamine treatment in patients with Late Onset Tay Sachs: an open label, extended pilot study.Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease.Dietary modifications in patients receiving miglustat.Haematopoietic Stem Cell Transplantation Arrests the Progression of Neurodegenerative Disease in Late-Onset Tay-Sachs Disease.Temporary Efficacy of Pyrimethamine in Juvenile-Onset Tay-Sachs Disease Caused by 2 Unreported HEXA Mutations in the Indian Population.
P2860
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P2860
Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment.
description
2009 nî lūn-bûn
@nan
2009年の論文
@ja
2009年学术文章
@wuu
2009年学术文章
@zh-cn
2009年学术文章
@zh-hans
2009年学术文章
@zh-my
2009年学术文章
@zh-sg
2009年學術文章
@yue
2009年學術文章
@zh
2009年學術文章
@zh-hant
name
Miglustat in late-onset Tay-Sa ...... months of extended treatment.
@en
Miglustat in late-onset Tay-Sa ...... months of extended treatment.
@nl
type
label
Miglustat in late-onset Tay-Sa ...... months of extended treatment.
@en
Miglustat in late-onset Tay-Sa ...... months of extended treatment.
@nl
prefLabel
Miglustat in late-onset Tay-Sa ...... months of extended treatment.
@en
Miglustat in late-onset Tay-Sa ...... months of extended treatment.
@nl
P2093
P2860
P1433
P1476
Miglustat in late-onset Tay-Sa ...... months of extended treatment.
@en
P2093
Barbara E Shapiro
Cécile Luzy
Edwin H Kolodny
Gregory M Pastores
John Gianutsos
P2860
P2888
P304
P356
10.1097/GIM.0B013E3181A1B5C5
P407
P577
2009-06-01T00:00:00Z
P6179
1018263260