Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase
about
Less Is More: Substrate Reduction Therapy for Lysosomal Storage DisordersThe Large Phenotypic Spectrum of Fabry Disease Requires Graduated Diagnosis and Personalized Therapy: A Meta-Analysis Can Help to Differentiate Missense MutationsCoformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry MiceQuantum dots and potential therapy for Krabbe's diseaseRecent advances and novel treatments for sphingolipidoses.Impact of immunosuppressive therapy on therapy-neutralizing antibodies in transplanted patients with Fabry disease.Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases.Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations.Fabry disease; early diagnosis improves prognosis but diagnosis is often delayed.Cell therapy for diverse central nervous system disorders: inherited metabolic diseases and autism.Glucosylceramide synthase inhibition with lucerastat lowers globotriaosylceramide and lysosome staining in cultured fibroblasts from Fabry patients with different mutation types
P2860
Q26744230-7EE5C782-44DF-43E6-87D8-6DB31B50F982Q36213022-D9DBD37E-D4DF-4CAB-8908-40CC157542DCQ36756784-8657757E-8F8E-4FA6-8E05-01D170727FCAQ37265520-7DED9307-2337-4CED-9790-CD60B80BFD07Q38635135-D5A05ECF-A089-4E4D-80F2-D28D76F29756Q38662053-116C9B72-3746-42AB-B96E-4F99B59F6220Q38797648-551E0814-1A3F-4CBE-8D5D-584A43033A60Q41671504-8366F8CD-E62B-4F34-9C65-219246DE708FQ41905133-171634B2-C87C-4B21-97A7-AFBD292ED68AQ49557884-616791FB-7A2D-40A4-B882-8501EF875FE6Q58794894-3D010E71-479B-42AA-8DF6-61B6CCEB67D7
P2860
Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase
description
2015 nî lūn-bûn
@nan
2015 թուականին հրատարակուած գիտական յօդուած
@hyw
2015 թվականին հրատարակված գիտական հոդված
@hy
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
name
Oral Migalastat HCl Leads to G ...... stered with Infused Agalsidase
@ast
Oral Migalastat HCl Leads to G ...... stered with Infused Agalsidase
@en
Oral Migalastat HCl Leads to G ...... stered with Infused Agalsidase
@nl
type
label
Oral Migalastat HCl Leads to G ...... stered with Infused Agalsidase
@ast
Oral Migalastat HCl Leads to G ...... stered with Infused Agalsidase
@en
Oral Migalastat HCl Leads to G ...... stered with Infused Agalsidase
@nl
prefLabel
Oral Migalastat HCl Leads to G ...... stered with Infused Agalsidase
@ast
Oral Migalastat HCl Leads to G ...... stered with Infused Agalsidase
@en
Oral Migalastat HCl Leads to G ...... stered with Infused Agalsidase
@nl
P2093
P2860
P3181
P1433
P1476
Oral Migalastat HCl Leads to G ...... stered with Infused Agalsidase
@en
P2093
Brandon A Wustman
Carrolee Barlow
David G Warnock
David J Lockhart
Francois Eyskens
Franklin K Johnson
John J Flanagan
Kenneth J Valenzano
Mark Thomas
P2860
P304
P3181
P356
10.1371/JOURNAL.PONE.0134341
P407
P577
2015-01-01T00:00:00Z