BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

183 related articles for article (PubMed ID: 21235442)

  • 1. Pompe disease: from new views on pathophysiology to innovative therapeutic strategies.
    Parenti G; Andria G
    Curr Pharm Biotechnol; 2011 Jun; 12(6):902-15. PubMed ID: 21235442
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Gene Therapy for Pompe Disease: The Time is now.
    Colella P; Mingozzi F
    Hum Gene Ther; 2019 Oct; 30(10):1245-1262. PubMed ID: 31298581
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Pompe Disease: New Developments in an Old Lysosomal Storage Disorder.
    Meena NK; Raben N
    Biomolecules; 2020 Sep; 10(9):. PubMed ID: 32962155
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Salmeterol enhances the cardiac response to gene therapy in Pompe disease.
    Han SO; Li S; Koeberl DD
    Mol Genet Metab; 2016 May; 118(1):35-40. PubMed ID: 27017193
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease.
    Yi H; Sun T; Armstrong D; Borneman S; Yang C; Austin S; Kishnani PS; Sun B
    J Mol Med (Berl); 2017 May; 95(5):513-521. PubMed ID: 28154884
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Advances in diagnosis and management of Pompe disease.
    Davison JE
    J Mother Child; 2020 Oct; 24(2):3-8. PubMed ID: 33554498
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
    Costa-Verdera H; Collaud F; Riling CR; Sellier P; Nordin JML; Preston GM; Cagin U; Fabregue J; Barral S; Moya-Nilges M; Krijnse-Locker J; van Wittenberghe L; Daniele N; Gjata B; Cosette J; Abad C; Simon-Sola M; Charles S; Li M; Crosariol M; Antrilli T; Quinn WJ; Gross DA; Boyer O; Anguela XM; Armour SM; Colella P; Ronzitti G; Mingozzi F
    Nat Commun; 2021 Nov; 12(1):6393. PubMed ID: 34737297
    [TBL] [Abstract][Full Text] [Related]  

  • 8. β2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease.
    Koeberl DD; Li S; Dai J; Thurberg BL; Bali D; Kishnani PS
    Mol Genet Metab; 2012 Feb; 105(2):221-7. PubMed ID: 22154081
    [TBL] [Abstract][Full Text] [Related]  

  • 9. New therapeutic approaches for Pompe disease: enzyme replacement therapy and beyond.
    Kishnani PS; Beckemeyer AA
    Pediatr Endocrinol Rev; 2014 Sep; 12 Suppl 1():114-24. PubMed ID: 25345093
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease.
    Han SO; Li S; Bird A; Koeberl D
    Hum Gene Ther; 2015 Nov; 26(11):743-50. PubMed ID: 26417913
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Improved efficacy of a next-generation ERT in murine Pompe disease.
    Xu S; Lun Y; Frascella M; Garcia A; Soska R; Nair A; Ponery AS; Schilling A; Feng J; Tuske S; Valle MCD; Martina JA; Ralston E; Gotschall R; Valenzano KJ; Puertollano R; Do HV; Raben N; Khanna R
    JCI Insight; 2019 Mar; 4(5):. PubMed ID: 30843882
    [TBL] [Abstract][Full Text] [Related]  

  • 12. The new era of Pompe disease: advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management.
    Kishnani PS; Beckemeyer AA; Mendelsohn NJ
    Am J Med Genet C Semin Med Genet; 2012 Feb; 160C(1):1-7. PubMed ID: 22253049
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Pharmacological Chaperone Therapy for Pompe Disease.
    Borie-Guichot M; Tran ML; Génisson Y; Ballereau S; Dehoux C
    Molecules; 2021 Nov; 26(23):. PubMed ID: 34885805
    [TBL] [Abstract][Full Text] [Related]  

  • 14. The role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease.
    Lacaná E; Yao LP; Pariser AR; Rosenberg AS
    Am J Med Genet C Semin Med Genet; 2012 Feb; 160C(1):30-9. PubMed ID: 22253234
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Abnormal mannose-6-phosphate receptor trafficking impairs recombinant alpha-glucosidase uptake in Pompe disease fibroblasts.
    Cardone M; Porto C; Tarallo A; Vicinanza M; Rossi B; Polishchuk E; Donaudy F; Andria G; De Matteis MA; Parenti G
    Pathogenetics; 2008 Dec; 1(1):6. PubMed ID: 19046416
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model.
    Xu F; Ding E; Liao SX; Migone F; Dai J; Schneider A; Serra D; Chen YT; Amalfitano A
    Gene Ther; 2004 Nov; 11(21):1590-8. PubMed ID: 15356673
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Infantile Pompe disease on ERT: update on clinical presentation, musculoskeletal management, and exercise considerations.
    Case LE; Beckemeyer AA; Kishnani PS
    Am J Med Genet C Semin Med Genet; 2012 Feb; 160C(1):69-79. PubMed ID: 22252989
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Pharmacological enhancement of α-glucosidase by the allosteric chaperone N-acetylcysteine.
    Porto C; Ferrara MC; Meli M; Acampora E; Avolio V; Rosa M; Cobucci-Ponzano B; Colombo G; Moracci M; Andria G; Parenti G
    Mol Ther; 2012 Dec; 20(12):2201-11. PubMed ID: 22990675
    [TBL] [Abstract][Full Text] [Related]  

  • 19. A Comprehensive Update on Late-Onset Pompe Disease.
    Labella B; Cotti Piccinelli S; Risi B; Caria F; Damioli S; Bertella E; Poli L; Padovani A; Filosto M
    Biomolecules; 2023 Aug; 13(9):. PubMed ID: 37759679
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Pompe disease: early diagnosis and early treatment make a difference.
    Chien YH; Hwu WL; Lee NC
    Pediatr Neonatol; 2013 Aug; 54(4):219-27. PubMed ID: 23632029
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 10.