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10.1186/s13023-018-0813-7 http://scihub22266oqcxt.onion/10.1186/s13023-018-0813-7 C5923014!5923014 !29703262     free     free     free Deprecated: Implicit conversion from float 225.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 225.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 225.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 225.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 225.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 225.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 259.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 259.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 259.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 259.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 259.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Warning: imagejpeg(C:\Inetpub\vhosts\kidney.de\httpdocs\phplern\29703262 .jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117       Orphanet+J+Rare+Dis 2018 ; 13 (1 ): 68 Nephropedia Template TP {{tp|p=29703262 |t=2018. Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial |pdf=|usr=}}{{29703262 }} gab.com Text Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial JO: Orphanet J Rare Dis http://www.kidney.de/pget.php?&tw=1&pmid=29703262 #FOAvip BACKGROUND: Fabry disease is frequently characterized by gastrointestinal symptoms, including diarrhea. Migalastat is an orally-administered small molecule approved to treat the symptoms of Fabry disease in patients with amenable mutations. METHODS: We evaluated minimal clinically important differences (MCID) in diarrhea based on the corresponding domain of the patient-reported Gastrointestinal Symptom Rating Scale (GSRS) in patients with Fabry disease and amenable mutations (N?=?50) treated with migalastat 150 mg every other day or placebo during the phase 3 FACETS trial (NCT00925301). RESULTS: After 6 months, significantly more patients receiving migalastat versus placebo experienced improvement in diarrhea based on a MCID of 0.33 (43% vs 11%; p?=?.02), including the subset with baseline diarrhea (71% vs 20%; p?=?.02). A decline in kidney peritubular capillary globotriaosylceramide inclusions correlated with diarrhea improvement; patients with a reduction >?0.1 were 5.6 times more likely to have an improvement in diarrhea than those without (p?=?.031). CONCLUSIONS: Migalastat was associated with a clinically meaningful improvement in diarrhea in patients with Fabry disease and amenable mutations. Reductions in kidney globotriaosylceramide may be a useful surrogate endpoint to predict clinical benefit with migalastat in patients with Fabry disease. TRIAL REGISTRATION: NCT00925301 ; June 19, 2009. Twit Text FOAVip Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial ????Orphanet J Rare Dis http://www.kidney.de/pget.php?&tw=1&pmid=29703262 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=29703262 #FOAvip English Wikipedia <ref>{{Cite pmid|29703262 }}</ref> Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial #MMPMID29703262 Schiffmann R ; Bichet DG ; Jovanovic A ; Hughes DA ; Giugliani R ; Feldt-Rasmussen U ; Shankar SP ; Barisoni L ; Colvin RB ; Jennette JC ; Holdbrook F ; Mulberg A ; Castelli JP ; Skuban N ; Barth JA ; Nicholls K Orphanet J Rare Dis 2018[Apr]; 13 (1 ): 68 PMID29703262 show ga BACKGROUND: Fabry disease is frequently characterized by gastrointestinal symptoms, including diarrhea. Migalastat is an orally-administered small molecule approved to treat the symptoms of Fabry disease in patients with amenable mutations. METHODS: We evaluated minimal clinically important differences (MCID) in diarrhea based on the corresponding domain of the patient-reported Gastrointestinal Symptom Rating Scale (GSRS) in patients with Fabry disease and amenable mutations (N?=?50) treated with migalastat 150 mg every other day or placebo during the phase 3 FACETS trial (NCT00925301). RESULTS: After 6 months, significantly more patients receiving migalastat versus placebo experienced improvement in diarrhea based on a MCID of 0.33 (43% vs 11%; p?=?.02), including the subset with baseline diarrhea (71% vs 20%; p?=?.02). A decline in kidney peritubular capillary globotriaosylceramide inclusions correlated with diarrhea improvement; patients with a reduction >?0.1 were 5.6 times more likely to have an improvement in diarrhea than those without (p?=?.031). CONCLUSIONS: Migalastat was associated with a clinically meaningful improvement in diarrhea in patients with Fabry disease and amenable mutations. Reductions in kidney globotriaosylceramide may be a useful surrogate endpoint to predict clinical benefit with migalastat in patients with Fabry disease. TRIAL REGISTRATION: NCT00925301 ; June 19, 2009. |1-Deoxynojirimycin/*analogs & derivatives/therapeutic use [MESH] |Adolescent [MESH] |Adult [MESH] |Aged [MESH] |Biomarkers/metabolism [MESH] |Diarrhea/*drug therapy [MESH] |Fabry Disease/*drug therapy/metabolism [MESH] |Female [MESH] |Humans [MESH] |Kidney/metabolism/pathology [MESH] |Male [MESH] |Middle Aged [MESH] |Mutation/genetics [MESH] |Trihexosylceramides [MESH]Migalastat improves diarrhea in patients with Fabry disease: clinical-(epmc).pdf DeepDyve Pubget Overpricing