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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.
Respiratory manifestations in patients with inherited metabolic diseases.
Santamaria F, Montella S, Mirra V, De Stefano S, Andria G, Parenti G. Santamaria F, et al. Eur Respir Rev. 2013 Dec;22(130):437-53. doi: 10.1183/09059180.00008012. Eur Respir Rev. 2013. PMID: 24293461 Review.
Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.
Ferla R, Claudiani P, Savarese M, Kozarsky K, Parini R, Scarpa M, Donati MA, Sorge G, Hopwood JJ, Parenti G, Fecarotta S, Nigro V, Sivri HS, Van Der Ploeg A, Andria G, Brunetti-Pierri N, Auricchio A. Ferla R, et al. Hum Gene Ther. 2015 Mar;26(3):145-52. doi: 10.1089/hum.2014.109. Hum Gene Ther. 2015. PMID: 25654180 Free PMC article. Clinical Trial.
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.
Vitamin E Improves Clinical Outcome of Patients Affected by Glycogen Storage Disease Type Ib.
Melis D, Minopoli G, Balivo F, Marcolongo P, Parini R, Paci S, Dionisi-Vici C, Della Casa R, Benedetti A, Andria G, Parenti G. Melis D, et al. JIMD Rep. 2016;25:39-45. doi: 10.1007/8904_2015_461. Epub 2015 Jun 30. JIMD Rep. 2016. PMID: 26122627 Free PMC article.
In seven patients G-CSF requirement decreased and the dose was reduced after the end of the study.In conclusion, our study demonstrated the efficacy of vitamin E supplementation. Vitamin E has evident advantages as compared to G-CSF, as it can be assumed orally, and …
In seven patients G-CSF requirement decreased and the dose was reduced after the end of the study.In conclusion, our study demonstrat …
Cutting Edge: Increased Autoimmunity Risk in Glycogen Storage Disease Type 1b Is Associated with a Reduced Engagement of Glycolysis in T Cells and an Impaired Regulatory T Cell Function.
Melis D, Carbone F, Minopoli G, La Rocca C, Perna F, De Rosa V, Galgani M, Andria G, Parenti G, Matarese G. Melis D, et al. Among authors: andria g. J Immunol. 2017 May 15;198(10):3803-3808. doi: 10.4049/jimmunol.1601946. Epub 2017 Apr 7. J Immunol. 2017. PMID: 28389590 Free PMC article.
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 Enhancement of Mutated α-Glucosidase Activity in Fibroblasts from Patients with Pompe Disease.
Parenti G, Zuppaldi A, Gabriela Pittis M, Rosaria Tuzzi M, Annunziata I, Meroni G, Porto C, Donaudy F, Rossi B, Rossi M, Filocamo M, Donati A, Bembi B, Ballabio A, Andria G. Parenti G, et al. Mol Ther. 2007 Mar;15(3):508-514. doi: 10.1038/sj.mt.6300074. Epub 2016 Dec 8. Mol Ther. 2007. PMID: 28182897
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.
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