Publications
Over the last several years there have been numerous papers published on the subject of Pompe disease and Pompe disease research. The following is a partial list (in chronological order) of these publications.
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September 2006
Hyaluronidase increases the biodistribution of acid a-14, glucosidase in the muscle of Pompe disease mice: An approach to enhance the efficacy of enzyme replacement therapy
Biochemical and Biophysical Research Communications, Courtesy of Jared Salbato
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September 2006
Chemical chaperones inprove transport and enhance stability of mutant a-glucosidases in glycogen storage disease type II
Molecular Genetics and Metabolism, Spetember 2006, Courtesy of Jared Salbato
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August 2006
Autophagy and Mistargeting of Therapeutic Enzyme in Skeletal Muscle in Pompe Disease
Molecular Therapy, August 2006, Courtesy of Jared Salbato
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April 2006
Dysfunction of Endocytic and Autophagic Pathways in a Lysosomal Storage Disease (Annals of Neurology, April 2006)
Annals of Neurology, April 2006, Courtesy of Jared Salbato
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January 2006
Characterization of Pre- and Post-Treatment Pathology After Enzyme Replacement Therapy for Pompe Disease
Laboratory Investigations, 2006, Courtesy of Jared Salbato
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July 2005
Evasion of Immune Responses to Introduced Human Acid alpha-Glucosidase by Liver-Restricted Expression in Glycogen Storage Disease Type II
Molecular Therapy, July 2005, Courtesy of Jared Salbato
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June 2005
Correction of Glycogen Storage Disease Type II by an Adeno-associated Virus Vector Containing a Muscle-Specific Promoter
Molecular Therapy, June 2005, courtesy of Jared Salbato
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June 2005
Disease severity in children and adults with Pompe disease related to age and disease duration
Neurology, June 2005, Courtesy of the IPA Web Site
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April 2005
Sustained correction of glycogen storage disease type II using adeno-associated virus serotype I vectors
Gene Therapy, April 2005, Courtesy of Jared Salbato
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January 2005
Impact of Humoral Immune Response on Distribution and Efficacy of Recombinant Adeno-Associated Virus-Derived Acid a-Glucosidase in a Model of Glycogen Storage Disease Type II
Human Gene Therapy, January 2005, courtesy of George Fox
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January 2005
Efficacy of an Adeno-associated Virus 8-Pseudotyped Vector in Glycogen Storage Disease Type II
Molecular Therapy, January 2005, courtesy of Monty Frost
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January 2005
Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients
Brain: A Journal of Neurology, January 2005, courtesy of the IPA Web Site
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November 2004
Conjugation of Mannose 6-Phosphate-containing Oligosaccharides to Acid a-Glucosidase Improves the Clearance of Glycogen in Pompe Mice
Journal of Biological Chemistry, November 2004
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November 2004
Late-onset Pompe disease primarily affects quality of life in physical health domains
Annals of Neurology, November 2004, courtesy of the IPA Web Site
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September 2004
Enhanced tissue delivery of acid alpha-glucosidase
Journal of Biological Chemistry, September 2004, courtesy of Jared Salbato
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August 2004
Lipoprotein Receptor Binding, Cellular Uptake, and Lysosomal Delivery of Fusions between the Receptor-associated Protein (RAP) and a-L-Iduronidase or Acid a-Glucosidase
Journal of Biological Chemistry, August 2004, courtesy of Jared Salbato
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May 2004
Pompe’s Disease. World Succes with a Setback: Laborious Production of the Medicine
Erasmus Monitor, April/May 2004, courtesy IPA Web Site
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May 2004
Long-Term Intravenous Treatment of Pompe Disease With Recombinant Human Alpha-Glucosidase From Milk
Pediatrics, May 2004, courtesy of Gezinus Wolters
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March 2004
Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease
Development, March 2004, courtesy of Jared Salbato
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March 2004
Virus Vector Delivery to Murine Diaphragm (Gene Therapy for Pompe's Disease a.o.)
Molecular Therapy, March 2004, courtesy Gezinus Wolters
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October 2003
Correction/mutation of acid a-D-glucosidase gene by modified single-stranded oligonucleotides: in vitro and in vivo studies
Gene Therapy, October 2003
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August 2003
The Natural Course of Infantile Pompe’s Disease: 20 Original Cases Compared With 133 Cases From the Literature
Pediatrics, August 2003
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July 2000
Recombinant human alpha-glucosidase from rabbit milk in Pompe patients
Lancet, July 2000
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February 1998
Clinical and Metabolic Correction of Pompe Disease by Enzyme Therapy in Acid Maltase–deficient Quail
Journal of Clinical Investigations, February 1998
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May 1997
Dietary treatment in late-onset acid maltase deficiency
European Journal of Pediatrics, 1997
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January 1936
J.C. Pompe's Thesis: Cardiomegalia Glycogenica
Courtesy of Gezinus Wolters' Homepage (The thesis itself is in Dutch, but there are French and English Summaries on the last few pages)