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Year Number of Results
2011 4
2012 3
2013 3
2014 3
2015 2
2020 0
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13 results
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Page 1
Lysosomal storage diseases: from pathophysiology to therapy.
Parenti G, Andria G, Ballabio A. Parenti G, et al. Annu Rev Med. 2015;66:471-86. doi: 10.1146/annurev-med-122313-085916. Annu Rev Med. 2015. PMID: 25587658 Review.
Pharmacological chaperone therapy for lysosomal storage diseases.
Parenti G, Moracci M, Fecarotta S, Andria G. Parenti G, et al. Future Med Chem. 2014 Jun;6(9):1031-45. doi: 10.4155/fmc.14.40. Future Med Chem. 2014. PMID: 25068986 Review.
Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders.
Parenti G, Andria G, Valenzano KJ. Parenti G, et al. Mol Ther. 2015 Jul;23(7):1138-1148. doi: 10.1038/mt.2015.62. Epub 2015 Apr 16. Mol Ther. 2015. PMID: 25881001 Free PMC article. Review.
Enzyme replacement therapy in patients with Fabry disease: state of the art and review of the literature.
Pisani A, Visciano B, Roux GD, Sabbatini M, Porto C, Parenti G, Imbriaco M. Pisani A, et al. Mol Genet Metab. 2012 Nov;107(3):267-75. doi: 10.1016/j.ymgme.2012.08.003. Epub 2012 Aug 11. Mol Genet Metab. 2012. PMID: 22963910 Review.
What else is in store for autophagy? Exocytosis of autolysosomes as a mechanism of TFEB-mediated cellular clearance in Pompe disease.
Feeney EJ, Spampanato C, Puertollano R, Ballabio A, Parenti G, Raben N. Feeney EJ, et al. Autophagy. 2013 Jul;9(7):1117-8. doi: 10.4161/auto.24920. Epub 2013 May 6. Autophagy. 2013. PMID: 23669057 Free PMC article.
Synergy between the pharmacological chaperone 1-deoxygalactonojirimycin and agalsidase alpha in cultured fibroblasts from patients with Fabry disease.
Pisani A, Porto C, Andria G, Parenti G. Pisani A, et al. J Inherit Metab Dis. 2014 Jan;37(1):145-6. doi: 10.1007/s10545-013-9641-z. Epub 2013 Aug 22. J Inherit Metab Dis. 2014. PMID: 23974650 No abstract available.
Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease.
Spampanato C, Feeney E, Li L, Cardone M, Lim JA, Annunziata F, Zare H, Polishchuk R, Puertollano R, Parenti G, Ballabio A, Raben N. Spampanato C, et al. EMBO Mol Med. 2013 May;5(5):691-706. doi: 10.1002/emmm.201202176. Epub 2013 Apr 18. EMBO Mol Med. 2013. PMID: 23606558 Free PMC article.
A chaperone enhances blood α-glucosidase activity in Pompe disease patients treated with enzyme replacement therapy.
Parenti G, Fecarotta S, la Marca G, Rossi B, Ascione S, Donati MA, Morandi LO, Ravaglia S, Pichiecchio A, Ombrone D, Sacchini M, Pasanisi MB, De Filippi P, Danesino C, Della Casa R, Romano A, Mollica C, Rosa M, Agovino T, Nusco E, Porto C, Andria G. Parenti G, et al. Mol Ther. 2014 Nov;22(11):2004-12. doi: 10.1038/mt.2014.138. Epub 2014 Jul 23. Mol Ther. 2014. PMID: 25052852 Free PMC article.
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. Porto C, et al. Mol Ther. 2012 Dec;20(12):2201-11. doi: 10.1038/mt.2012.152. Epub 2012 Sep 18. Mol Ther. 2012. PMID: 22990675 Free PMC article.
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