Compounds > 1-deoxynojirimycin
Page last updated: 2024-08-23

1-deoxynojirimycin and Glycogen Storage Disease Type II

1-deoxynojirimycin has been researched along with Glycogen Storage Disease Type II in 17 studies

Research

Studies (17)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's1 (5.88)18.2507
2000's4 (23.53)29.6817
2010's9 (52.94)24.3611
2020's3 (17.65)2.80

Authors

AuthorsStudies
Fleet, GWJ; Jia, YM; Kanekiyo, U; Kato, A; Kishida, M; Li, YX; Lu, TT; Nakagome, I; Nash, RJ; Shinzawa, K; Tanaka, N; Yoshida, T; Yoshimura, K; Yu, CY1
Bratkovic, D; Byrne, BJ; Castelli, J; Díaz-Manera, J; Goldman, M; Jiang, H; Kishnani, PS; Kuchipudi, S; Laforêt, P; Mozaffar, T; Roberts, M; Schoser, B; Sitaraman, S; Toscano, A; van der Ploeg, AT1
Blair, HA1
Adera, M; Barlow, C; Barth, J; Benjamin, E; Boudes, P; Byrne, B; Dasouki, M; Dimachkie, MM; Feng, J; Finanger, E; Flanagan, JJ; Goker-Alpan, O; Guter, KA; Johnson, FK; Khanna, R; Kishnani, P; Laforet, P; Lazauskas, R; Levine, T; Lockhart, DJ; Mozaffar, T; Pervaiz, MA; Roberts, M; Sitaraman, S; Sivakumar, K; Tarnopolsky, M; Valenzano, KJ1
Cobucci-Ponzano, B; D'Alonzo, D; De Fenza, M; Guaragna, A; Huebecker, M; Iacono, R; Moracci, M; Palumbo, G; Parenti, G; Platt, F; Porto, C; Priestman, DA1
Do, HV; Feng, J; Frascella, M; Garcia, A; Gotschall, R; Khanna, R; Lun, Y; Martina, JA; Nair, A; Ponery, AS; Puertollano, R; Raben, N; Ralston, E; Schilling, A; Soska, R; Tuske, S; Valenzano, KJ; Valle, MCD; Xu, S1
Brignol, N; Dhulipala, R; Do, HV; Feng, J; Frascella, M; Garcia, A; Khanna, R; Lockhart, DJ; Lun, Y; Pellegrino, LJ; Powe, AC; Soska, R; Toth, MJ; Valenzano, KJ; Wustman, BA; Xu, S1
Agovino, T; Andria, G; Ascione, S; Danesino, C; De Filippi, P; Della Casa, R; Donati, MA; Fecarotta, S; la Marca, G; Mollica, C; Morandi, LO; Nusco, E; Ombrone, D; Parenti, G; Pasanisi, MB; Pichiecchio, A; Porto, C; Ravaglia, S; Romano, A; Rosa, M; Rossi, B; Sacchini, M1
Beller, M; Giese, AK; Gläser, A; Lukas, J; Pockrandt, AM; Pohlers, S; Rolfs, A; Runge, F; Seemann, S; Sharif, M; Zheng, C1
Andreotti, G; Cubellis, MV; Monticelli, M1
Andria, G; Barone, MV; Cardone, M; Fontana, F; Parenti, G; Porto, C; Rossi, B; Tarallo, A; Tuzzi, MR1
Andria, G; Benjamin, E; Cubellis, MV; Do, HV; Donaudy, F; Flanagan, JJ; Fontana, F; Lockhart, DJ; Mascioli, K; Parenti, G; Porto, C; Rossi, B; Tang, K; Tuzzi, MR; Valenzano, KJ; Wu, X1
Aoki, K; Eto, Y; Ida, H; Kaneshiro, E; Kawagoe, S; Kobayashi, H; Ohashi, T; Shimada, Y; Shimizu, H1
Feng, J; Flanagan, JJ; Frascella, M; Guillen, D; Khanna, R; Lockhart, DJ; Lun, Y; Pellegrino, LJ; Soska, R; Valenzano, KJ1
Kroos, MA; Okumiya, T; Reuser, AJ; Takeuchi, H; Van der Ploeg, AT; Vliet, LV1
Aikawa, S; Edmunds, T; Fujishima, K; Ikekita, M; Itoh, K; Iwamoto, K; Kawashima, I; Kobayashi, T; Matsuzawa, F; Ohno, K; Ohyanagi, N; Sakuraba, H; Sugawara, K; Suzuki, T; Tajima, Y; Togawa, T; Tsuji, D; Yoshimizu, M1
Creutzfeldt, W; Lamberts, R; Lembcke, B; Wöhler, J1

Reviews

1 review(s) available for 1-deoxynojirimycin and Glycogen Storage Disease Type II

ArticleYear
Cipaglucosidase Alfa: First Approval.
    Drugs, 2023, Volume: 83, Issue:8

    Topics: 1-Deoxynojirimycin; Adult; Enzyme Replacement Therapy; Glycogen; Glycogen Storage Disease Type II; Humans

2023

Trials

2 trial(s) available for 1-deoxynojirimycin and Glycogen Storage Disease Type II

ArticleYear
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
    The Lancet. Neurology, 2021, Volume: 20, Issue:12

    Topics: 1-Deoxynojirimycin; Adolescent; alpha-Glucosidases; Double-Blind Method; Glycogen Storage Disease Type II; Humans; Treatment Outcome

2021
Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α.
    Molecular therapy : the journal of the American Society of Gene Therapy, 2017, 05-03, Volume: 25, Issue:5

    Topics: 1-Deoxynojirimycin; Administration, Oral; Adult; alpha-Glucosidases; Drug Administration Schedule; Drug Synergism; Drug Therapy, Combination; Enzyme Replacement Therapy; Female; Glycogen Storage Disease Type II; Humans; Infusions, Intravenous; Lysosomes; Male; Middle Aged; Muscle, Skeletal; Patient Safety; Treatment Outcome

2017

Other Studies

14 other study(ies) available for 1-deoxynojirimycin and Glycogen Storage Disease Type II

ArticleYear
5-
    Journal of medicinal chemistry, 2022, 02-10, Volume: 65, Issue:3

    Topics: 1-Deoxynojirimycin; Alkylation; alpha-Glucosidases; Enzyme Inhibitors; Fibroblasts; Glycogen Storage Disease Type II; Humans; Molecular Dynamics Simulation; Molecular Structure; Mutation; Protein Conformation; Protein Stability; Recombinant Proteins

2022
N-Butyl-l-deoxynojirimycin (l-NBDNJ): Synthesis of an Allosteric Enhancer of α-Glucosidase Activity for the Treatment of Pompe Disease.
    Journal of medicinal chemistry, 2017, 12-14, Volume: 60, Issue:23

    Topics: 1-Deoxynojirimycin; Allosteric Regulation; alpha-Glucosidases; Cell Line; Enzyme Activation; Enzyme Inhibitors; Fibroblasts; Glycogen Storage Disease Type II; Humans; Lysosomes; Models, Molecular; Stereoisomerism

2017
Improved efficacy of a next-generation ERT in murine Pompe disease.
    JCI insight, 2019, 03-07, Volume: 4, Issue:5

    Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Animals; Disease Models, Animal; Enzyme Replacement Therapy; Female; Glycogen; Glycogen Storage Disease Type II; Humans; Lysosomes; Male; Mannosephosphates; Mice; Mice, Knockout; Muscle, Skeletal; Rats; Rats, Sprague-Dawley

2019
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.
    PloS one, 2014, Volume: 9, Issue:7

    Topics: 1-Deoxynojirimycin; Administration, Oral; Animals; Biocatalysis; Biological Availability; Chlorocebus aethiops; COS Cells; Disease Models, Animal; Endoplasmic Reticulum; Enzyme Stability; Gene Knockout Techniques; Glucan 1,4-alpha-Glucosidase; Glycogen; Glycogen Storage Disease Type II; Humans; Isoenzymes; Lysosomes; Mice; Mice, Transgenic; Mutant Proteins; Mutation; Protein Transport; Proteolysis

2014
A chaperone enhances blood α-glucosidase activity in Pompe disease patients treated with enzyme replacement therapy.
    Molecular therapy : the journal of the American Society of Gene Therapy, 2014, Volume: 22, Issue:11

    Topics: 1-Deoxynojirimycin; Adolescent; Adult; alpha-Glucosidases; Animals; Child; Child, Preschool; Disease Models, Animal; Dried Blood Spot Testing; Drug Synergism; Enzyme Replacement Therapy; Enzyme Stability; Female; Glycogen Storage Disease Type II; Glycoside Hydrolase Inhibitors; Humans; Male; Mice; Middle Aged; Young Adult

2014
Enzyme enhancers for the treatment of Fabry and Pompe disease.
    Molecular therapy : the journal of the American Society of Gene Therapy, 2015, Volume: 23, Issue:3

    Topics: 1-Deoxynojirimycin; Acetylcysteine; alpha-Galactosidase; alpha-Glucosidases; Ambroxol; Bezafibrate; Enzyme Activators; Fabry Disease; Gene Expression; Glycogen Storage Disease Type II; HEK293 Cells; Humans; Leupeptins; Lysosomes; Pioglitazone; Plasmids; Proteasome Endopeptidase Complex; Proteasome Inhibitors; Protein Stability; Recombinant Proteins; Thiazolidinediones; Transfection

2015
Looking for protein stabilizing drugs with thermal shift assay.
    Drug testing and analysis, 2015, Volume: 7, Issue:9

    Topics: 1-Deoxynojirimycin; alpha-Galactosidase; Drug Evaluation, Preclinical; Enzyme Stability; Fabry Disease; Glycogen Storage Disease Type II; High-Throughput Screening Assays; Humans; Temperature

2015
The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts.
    Molecular therapy : the journal of the American Society of Gene Therapy, 2009, Volume: 17, Issue:6

    Topics: 1-Deoxynojirimycin; alpha-Galactosidase; Animals; Biological Transport; Blotting, Western; Cell Line; Drug Stability; Enzyme Inhibitors; Fibroblasts; Glycogen Storage Disease Type II; Humans; Lysosomes; Mice; Microscopy, Confocal

2009
The pharmacological chaperone 1-deoxynojirimycin increases the activity and lysosomal trafficking of multiple mutant forms of acid alpha-glucosidase.
    Human mutation, 2009, Volume: 30, Issue:12

    Topics: 1-Deoxynojirimycin; Adolescent; Adult; alpha-Glucosidases; Animals; Chlorocebus aethiops; COS Cells; Enzyme Stability; Fibroblasts; Glycogen Storage Disease Type II; Humans; Infant; Lysosomes; Models, Molecular; Mutant Proteins; Protein Structure, Secondary; Protein Transport; Recombinant Proteins

2009
Endoplasmic reticulum stress induces autophagy through activation of p38 MAPK in fibroblasts from Pompe disease patients carrying c.546G>T mutation.
    Molecular genetics and metabolism, 2011, Volume: 104, Issue:4

    Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Autophagy; Cells, Cultured; DNA Mutational Analysis; Endoplasmic Reticulum Stress; Enzyme Activation; Enzyme Activators; Fibroblasts; Glycogen Storage Disease Type II; MAP Kinase Signaling System; p38 Mitogen-Activated Protein Kinases; Phosphoproteins; Phosphorylation; Point Mutation; Protein Folding; Protein Isoforms; Unfolded Protein Response

2011
The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease.
    PloS one, 2012, Volume: 7, Issue:7

    Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Animals; Buffers; Disease Models, Animal; Enzyme Activation; Enzyme Stability; Glycogen; Glycogen Storage Disease Type II; Half-Life; Humans; Mice; Mice, Knockout; Protein Denaturation; Rats; Recombinant Proteins

2012
Chemical chaperones improve transport and enhance stability of mutant alpha-glucosidases in glycogen storage disease type II.
    Molecular genetics and metabolism, 2007, Volume: 90, Issue:1

    Topics: 1-Deoxynojirimycin; Adolescent; Adult; alpha-Glucosidases; Amino Acid Substitution; Animals; Cells, Cultured; Child, Preschool; Chlorocebus aethiops; COS Cells; Enzyme Inhibitors; Enzyme Stability; Glycogen Storage Disease Type II; Glycoside Hydrolase Inhibitors; Humans; Infant; Molecular Chaperones; Protein Transport; Sequence Deletion

2007
Binding parameters and thermodynamics of the interaction of imino sugars with a recombinant human acid alpha-glucosidase (alglucosidase alfa): insight into the complex formation mechanism.
    Clinica chimica acta; international journal of clinical chemistry, 2008, Volume: 391, Issue:1-2

    Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Binding Sites; Catalytic Domain; Drug Interactions; Glycogen Storage Disease Type II; Glycoside Hydrolase Inhibitors; Humans; Imino Sugars; Models, Molecular; Recombinant Proteins; Thermodynamics

2008
Lysosomal storage of glycogen as a sequel of alpha-glucosidase inhibition by the absorbed deoxynojirimycin derivative emiglitate (BAYo1248). A drug-induced pattern of hepatic glycogen storage mimicking Pompe's disease (glycogenosis type II).
    Research in experimental medicine. Zeitschrift fur die gesamte experimentelle Medizin einschliesslich experimenteller Chirurgie, 1991, Volume: 191, Issue:6

    Topics: 1-Deoxynojirimycin; Acarbose; Animals; Female; Glucosamine; Glycogen Storage Disease Type II; Glycoside Hydrolase Inhibitors; Imino Pyranoses; Liver; Liver Glycogen; Lysosomes; Microscopy, Electron; Rats; Rats, Inbred Strains; Trisaccharides

1991