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10.1016/j.jpeds.2015.11.079 http://scihub22266oqcxt.onion/10.1016/j.jpeds.2015.11.079 C4769976!4769976!26787381     free     free     free Deprecated: Implicit conversion from float 233.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 233.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 233.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       J+Pediatr 2016 ; 170 (ä): 278-287.e4 Nephropedia Template TP {{tp|p=26787381|t=2016. A prospective natural history study of Mucopolysaccharidosis Type IIIA |pdf=|usr=}}{{26787381}} gab.com Text A prospective natural history study of Mucopolysaccharidosis Type IIIA JO: J Pediatr http://www.kidney.de/pget.php?&tw=1&pmid=26787381 #FOAvip Objectives: To characterize the clinical course of mucopolysaccharidosis type IIIA (MPS IIIA), and identified potential endpoints for future treatment trials. Study design: Children with a confirmed diagnosis of MPS IIIA, functioning above a developmental age of 1 year, were followed for up to 2 years. Cognitive status and brain atrophy were assessed by standardized tests and volumetric MRI, respectively. Liver and spleen volumes, CSF and urine biomarker levels were measured. Results: Twenty-five children, from 1.1 to 18.4 years old, were enrolled, and 24 followed for at least 12 months. 19 exhibited a rapidly progressing form of MPS IIIA (RP), and 5, a more slowly progressing form (SP). Children with RP plateaued in development by 30 months, followed by rapid regression after 40 to 50 months. Cognitive developmental quotients (DQ) in patients with RP showed consistent steep declines associated with progressive cortical gray matter atrophy. Children with SP had a similar but more prolonged course. Liver and spleen volumes were approximately double normal size, and CSF and urine HS levels were elevated and relatively constant over time. Conclusions: DQ and cortical gray matter volumes are sensitive markers of disease progression in MPS IIIA, and may have utility as clinical endpoints in treatment trials. For optimal outcomes, treatment may need to be instituted in children before the onset of steep cognitive decline and brain atrophy. Twit Text FOAVip A prospective natural history study of Mucopolysaccharidosis Type IIIA ????J Pediatr http://www.kidney.de/pget.php?&tw=1&pmid=26787381 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=26787381 #FOAvip English Wikipedia <ref>{{Cite pmid|26787381}}</ref> A prospective natural history study of Mucopolysaccharidosis Type IIIA #MMPMID26787381 Shapiro EG; Nestrasil I; Delaney KA; Rudser K; Kovac V; Nair N; Richard CW; Haslett P; Whitley CB J Pediatr 2016[Mar]; 170 (ä): 278-287.e4 PMID26787381show ga Objectives: To characterize the clinical course of mucopolysaccharidosis type IIIA (MPS IIIA), and identified potential endpoints for future treatment trials. Study design: Children with a confirmed diagnosis of MPS IIIA, functioning above a developmental age of 1 year, were followed for up to 2 years. Cognitive status and brain atrophy were assessed by standardized tests and volumetric MRI, respectively. Liver and spleen volumes, CSF and urine biomarker levels were measured. Results: Twenty-five children, from 1.1 to 18.4 years old, were enrolled, and 24 followed for at least 12 months. 19 exhibited a rapidly progressing form of MPS IIIA (RP), and 5, a more slowly progressing form (SP). Children with RP plateaued in development by 30 months, followed by rapid regression after 40 to 50 months. Cognitive developmental quotients (DQ) in patients with RP showed consistent steep declines associated with progressive cortical gray matter atrophy. Children with SP had a similar but more prolonged course. Liver and spleen volumes were approximately double normal size, and CSF and urine HS levels were elevated and relatively constant over time. Conclusions: DQ and cortical gray matter volumes are sensitive markers of disease progression in MPS IIIA, and may have utility as clinical endpoints in treatment trials. For optimal outcomes, treatment may need to be instituted in children before the onset of steep cognitive decline and brain atrophy. äA prospective natural history study of Mucopolysaccharidosis Type IIIA(epmc).pdf DeepDyve Pubget Overpricing