The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts
about
Targeted approaches to induce immune tolerance for Pompe disease therapyNew insights into therapeutic options for Pompe diseaseNew therapeutic approaches for Krabbe disease: The potential of pharmacological chaperonesA rapid and sensitive method for measuring N-acetylglucosaminidase activity in cultured cellsThe pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe diseaseIdentification and characterization of ambroxol as an enzyme enhancement agent for Gaucher diseasePompe disease: from pathophysiology to therapy and back againRapid assembly of a library of lipophilic iminosugars via the thiol-ene reaction yields promising pharmacological chaperones for the treatment of Gaucher diseaseIminosugars as therapeutic agents: recent advances and promising trends.Therapeutic approaches for lysosomal storage diseasesThe pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease.Fabry disease - current treatment and new drug development.Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.Pompe disease: literature review and case seriesPharmacological chaperone therapy for lysosomal storage diseases.Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease.A chaperone enhances blood α-glucosidase activity in Pompe disease patients treated with enzyme replacement therapy.Azasugar inhibitors as pharmacological chaperones for Krabbe diseasePharmacological chaperone therapy for Fabry diseaseDiagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert groupHypertransaminasemia and fatal lung disease: a case report.Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry MiceTranscription factor EB (TFEB) is a new therapeutic target for Pompe diseaseLow-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics.Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders.Emerging drugs for lysosomal storage diseases.Pharmacological small molecules for the treatment of lysosomal storage disorders.Pharmacological chaperone therapy for Gaucher disease: a patent review.Treatment options for lysosomal storage disorders: developing insights.Glucocerebrosidase inhibitors for the treatment of Gaucher disease.Pharmacological chaperones for enzyme enhancement therapy in genetic diseases.Treatment of lysosomal storage disorders: successes and challenges.Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders.Pharmaceutical Chaperones and Proteostasis Regulators in the Therapy of Lysosomal Storage Disorders: Current Perspective and Future Promises.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Synthesis of lipophilic 1-deoxygalactonojirimycin derivatives as D-galactosidase inhibitors.Alglucosidase alfa: Long term use in the treatment of patients with Pompe disease.Pharmacological enhancement of α-glucosidase by the allosteric chaperone N-acetylcysteine.Co-administration with the pharmacological chaperone AT1001 increases recombinant human α-galactosidase A tissue uptake and improves substrate reduction in Fabry mice.
P2860
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P2860
The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts
description
2009 nî lūn-bûn
@nan
2009年の論文
@ja
2009年論文
@yue
2009年論文
@zh-hant
2009年論文
@zh-hk
2009年論文
@zh-mo
2009年論文
@zh-tw
2009年论文
@wuu
2009年论文
@zh
2009年论文
@zh-cn
name
The pharmacological chaperone ...... y in Pompe disease fibroblasts
@en
The pharmacological chaperone ...... in Pompe disease fibroblasts.
@nl
type
label
The pharmacological chaperone ...... y in Pompe disease fibroblasts
@en
The pharmacological chaperone ...... in Pompe disease fibroblasts.
@nl
prefLabel
The pharmacological chaperone ...... y in Pompe disease fibroblasts
@en
The pharmacological chaperone ...... in Pompe disease fibroblasts.
@nl
P2093
P2860
P921
P356
P1433
P1476
The pharmacological chaperone ...... y in Pompe disease fibroblasts
@en
P2093
Antonietta Tarallo
Barbara Rossi
Caterina Porto
Federica Fontana
Generoso Andria
Giancarlo Parenti
Maria Rosaria Tuzzi
Monica Cardone
P2860
P304
P356
10.1038/MT.2009.53
P577
2009-03-17T00:00:00Z