The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts - Wikidata - wikimedia - TriplyDB

The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts

The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts

http://www.wikidata.org/entity/Q39508157

about

Targeted approaches to induce immune tolerance for Pompe disease therapy New insights into therapeutic options for Pompe disease New therapeutic approaches for Krabbe disease: The potential of pharmacological chaperones A rapid and sensitive method for measuring N-acetylglucosaminidase activity in cultured cells The 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 disease Identification and characterization of ambroxol as an enzyme enhancement agent for Gaucher disease Pompe disease: from pathophysiology to therapy and back again Rapid assembly of a library of lipophilic iminosugars via the thiol-ene reaction yields promising pharmacological chaperones for the treatment of Gaucher disease Iminosugars as therapeutic agents: recent advances and promising trends.Therapeutic approaches for lysosomal storage diseases The 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 series Pharmacological 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 disease Pharmacological chaperone therapy for Fabry disease Diagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert group Hypertransaminasemia 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 Mice Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease Low-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.

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P2860

The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts

http://www.wikidata.org/entity/Q39508157

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The pharmacological chaperone ...... y in Pompe disease fibroblasts

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The pharmacological chaperone ...... y in Pompe disease fibroblasts

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Antonietta Tarallo

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Barbara Rossi

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Caterina Porto

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Federica Fontana

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Generoso Andria

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Giancarlo Parenti

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Maria Rosaria Tuzzi

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Monica Cardone

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P2860

Mutation profile of the GAA gene in 40 Italian patients with late onset glycogen storage disease type II

Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II

Structural and functional changes of lysosomal acid alpha-glucosidase during intracellular transport and maturation

Pyrimethamine as a potential pharmacological chaperone for late-onset forms of GM2 gangliosidosis.

Pharmacological enhancement of beta-hexosaminidase activity in fibroblasts from adult Tay-Sachs and Sandhoff Patients.

Role of autophagy in the pathogenesis of Pompe disease.

Imino sugar inhibitors for treating the lysosomal glycosphingolipidoses.

Potential efficacy of enzyme replacement and substrate reduction therapy in three siblings with Gaucher disease type III.

Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.

The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature.

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964-971

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10.1038/MT.2009.53

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6

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17

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Maria Vittoria Barone

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24207848

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19293774

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molecular chaperones

Enzyme replacement therapy

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2835191

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