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Pediatric Rehabilitation
|
June 19, 2004
Physical performance testing in mucopolysaccharidosis I: a pilot study
Helene M Dumas, Maria A Fragala, Stephen M Haley, et al.
The American Journal of Surgical Pathology
|
May 23, 2012
Liver and skin histopathology in adults with acid sphingomyelinase deficiency (Niemann-Pick disease type B)
Beth L Thurberg, Melissa P Wasserstein, Thomas Schiano, et al.
Molecular Genetics and Metabolism Reports
|
January 20, 2023
Neuropsychological endpoints for clinical trials in methylmalonic acidemia and propionic acidemia: A pilot study
Kimberly A Chapman, Devon MacEachern, Gerald F Cox, et al.
The American Journal of Surgical Pathology
|
June 25, 2016
Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency
Beth L Thurberg, Melissa P Wasserstein, Simon A Jones, et al.
Developmental Medicine and Child Neurology
|
June 20, 2006
A physical performance measure for individuals with mucopolysaccharidosis type I
Stephen M Haley, Maria A Fragala Pinkham, Helene M Dumas, et al.
Molecular Genetics and Metabolism
|
January 15, 2014
Lyso-sphingomyelin is elevated in dried blood spots of Niemann-Pick B patients
Wei-Lien Chuang, Joshua Pacheco, Samantha Cooper, et al.
Blood Cells, Molecules & Diseases
|
January 28, 2017
A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency, timing, and duration
M Judith Peterschmitt, Gerald F Cox, Jennifer Ibrahim, et al.
European Journal of Pediatrics
|
January 12, 2012
Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry
Kristin D'Aco, Lisa Underhill, Lakshmi Rangachari, et al.
American Journal of Medical Genetics. Part A
|
November 15, 2007
Encephalocraniocutaneous lipomatosis accompanied by the formation of bone cysts: Harboring clues to pathogenesis?
Ute Moog, Filip Roelens, Geert R Mortier, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
April 4, 2015
Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency)
Margaret M McGovern, Melissa P Wasserstein, Brian Kirmse, et al.
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of 8
Search research articles
Search
Showing results (21-30 of 75) with videos related to
Sort By:
Page
of 8
Pediatric Rehabilitation
|
June 19, 2004
Physical performance testing in mucopolysaccharidosis I: a pilot study
Helene M Dumas, Maria A Fragala, Stephen M Haley, et al.
The American Journal of Surgical Pathology
|
May 23, 2012
Liver and skin histopathology in adults with acid sphingomyelinase deficiency (Niemann-Pick disease type B)
Beth L Thurberg, Melissa P Wasserstein, Thomas Schiano, et al.
Molecular Genetics and Metabolism Reports
|
January 20, 2023
Neuropsychological endpoints for clinical trials in methylmalonic acidemia and propionic acidemia: A pilot study
Kimberly A Chapman, Devon MacEachern, Gerald F Cox, et al.
The American Journal of Surgical Pathology
|
June 25, 2016
Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency
Beth L Thurberg, Melissa P Wasserstein, Simon A Jones, et al.
Developmental Medicine and Child Neurology
|
June 20, 2006
A physical performance measure for individuals with mucopolysaccharidosis type I
Stephen M Haley, Maria A Fragala Pinkham, Helene M Dumas, et al.
Molecular Genetics and Metabolism
|
January 15, 2014
Lyso-sphingomyelin is elevated in dried blood spots of Niemann-Pick B patients
Wei-Lien Chuang, Joshua Pacheco, Samantha Cooper, et al.
Blood Cells, Molecules & Diseases
|
January 28, 2017
A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency, timing, and duration
M Judith Peterschmitt, Gerald F Cox, Jennifer Ibrahim, et al.
European Journal of Pediatrics
|
January 12, 2012
Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry
Kristin D'Aco, Lisa Underhill, Lakshmi Rangachari, et al.
American Journal of Medical Genetics. Part A
|
November 15, 2007
Encephalocraniocutaneous lipomatosis accompanied by the formation of bone cysts: Harboring clues to pathogenesis?
Ute Moog, Filip Roelens, Geert R Mortier, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
April 4, 2015
Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency)
Margaret M McGovern, Melissa P Wasserstein, Brian Kirmse, et al.
Page
of 8