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10.1038/mt.2014.138 http://scihub22266oqcxt.onion/10.1038/mt.2014.138 C4429731!4429731!25052852     free     free     free Deprecated: Implicit conversion from float 227.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 227.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 227.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 227.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 227.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 227.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 261.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 261.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       Mol+Ther 2014 ; 22 (11): 2004-12 Nephropedia Template TP {{tp|p=25052852|t=2014. A Chaperone Enhances Blood ?-Glucosidase Activity in Pompe Disease Patients Treated With Enzyme Replacement Therapy |pdf=|usr=}}{{25052852}} gab.com Text A Chaperone Enhances Blood -Glucosidase Activity in Pompe Disease Patients Treated With Enzyme Replacement Therapy JO: Mol Ther http://www.kidney.de/pget.php?&tw=1&pmid=25052852 #FOAvip Enzyme replacement therapy is currently the only approved treatment for Pompe disease, due to acid ?-glucosidase deficiency. Clinical efficacy of this approach is variable, and more effective therapies are needed. We showed in preclinical studies that chaperones stabilize the recombinant enzyme used for enzyme replacement therapy. Here, we evaluated the effects of a combination of enzyme therapy and a chaperone on ?-glucosidase activity in Pompe disease patients. ?-Glucosidase activity was analyzed by tandem-mass spectrometry in dried blood spots from patients treated with enzyme replacement therapy, either alone or in combination with the chaperone N-butyldeoxynojirimycin given at the time of the enzyme infusion. Thirteen patients with different presentations (3 infantile-onset, 10 late-onset) were enrolled. In 11 patients, the combination treatment resulted in ?-glucosidase activities greater than 1.85-fold the activities with enzyme replacement therapy alone. In the whole patient population, ?-glucosidase activity was significantly increased at 12 hours (2.19-fold, P = 0.002), 24 hours (6.07-fold, P = 0.001), and 36 hours (3.95-fold, P = 0.003). The areas under the curve were also significantly increased (6.78-fold, P = 0.002). These results suggest improved stability of recombinant ?-glucosidase in blood in the presence of the chaperone. Twit Text FOAVip A Chaperone Enhances Blood -Glucosidase Activity in Pompe Disease Patients Treated With Enzyme Replacement Therapy ????Mol Ther http://www.kidney.de/pget.php?&tw=1&pmid=25052852 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=25052852 #FOAvip English Wikipedia <ref>{{Cite pmid|25052852}}</ref> A Chaperone Enhances Blood ?-Glucosidase Activity in Pompe Disease Patients Treated With Enzyme Replacement Therapy #MMPMID25052852 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 Mol Ther 2014[Nov]; 22 (11): 2004-12 PMID25052852show ga Enzyme replacement therapy is currently the only approved treatment for Pompe disease, due to acid ?-glucosidase deficiency. Clinical efficacy of this approach is variable, and more effective therapies are needed. We showed in preclinical studies that chaperones stabilize the recombinant enzyme used for enzyme replacement therapy. Here, we evaluated the effects of a combination of enzyme therapy and a chaperone on ?-glucosidase activity in Pompe disease patients. ?-Glucosidase activity was analyzed by tandem-mass spectrometry in dried blood spots from patients treated with enzyme replacement therapy, either alone or in combination with the chaperone N-butyldeoxynojirimycin given at the time of the enzyme infusion. Thirteen patients with different presentations (3 infantile-onset, 10 late-onset) were enrolled. In 11 patients, the combination treatment resulted in ?-glucosidase activities greater than 1.85-fold the activities with enzyme replacement therapy alone. In the whole patient population, ?-glucosidase activity was significantly increased at 12 hours (2.19-fold, P = 0.002), 24 hours (6.07-fold, P = 0.001), and 36 hours (3.95-fold, P = 0.003). The areas under the curve were also significantly increased (6.78-fold, P = 0.002). These results suggest improved stability of recombinant ?-glucosidase in blood in the presence of the chaperone. äA Chaperone Enhances Blood ?-Glucosidase Activity in Pompe Disease Pat(epmc).pdf DeepDyve Pubget Overpricing