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Over the last several years, there have been several publications written in the field of Pompe's Disease research. The following publications, in chronological order, are a partial compilation of those that are the most relevant to Pompe's Disease research. July 2000 -- Recombinant human alpha-glucosidase from rabbit milk in Pompe patients (Lancet, July 2000) March 2001 -- Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial (Genetics in Medicine, March 2001) April 2001 -- Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk (Journal of Inherited Metabolic Disorders, April 2001) August 2003 -- The Natural Course of Infantile Pompe’s Disease: 20 Original Cases Compared With 133 Cases From the Literature (Pediatrics, August 2003) March 2004 -- Virus Vector Delivery to Murine Diaphragm (Gene Therapy for Pompe's Disease a.o.)(Molecular Therapy, March 2004)
April 2004 -- Enzyme Replacement Therapy in Late-Onset Pompe ’s Disease: A Three-Year Follow-up (Annals of Neurology, April 2004)
May 2004 -- Long-Term Intravenous Treatment of Pompe Disease With Recombinant Human Alpha-Glucosidase From Milk (Pediatrics, May 2004)
May 2004 -- Pompe’s Disease. World Success with a Setback: Laborious Production of the Medicine (Erasmus Monitor, April/May 2004)
November 2004 -- Late-onset Pompe disease primarily affects quality of life in physical health domains (Annals of Neurology, November 2004) January 2005 -- Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients (Brain: A Journal of Neurology, January 2005) March 2005 -- Muscle-Targeted Gene Therapy Reverses Rare Muscular Dystrophy in Mice (Molecular Therapy, March 2005) June 2005 -- Disease severity in children and adults with Pompe disease related to age and disease duration (Neurology, June 2005) May 2006 -- Physical Therapy Management of Pompe Disease (Genetics in Medicine, May 2006) May 2006 -- Pompe Disease Diagnosis and Management Guide (Genetics in Medicine, May 2006) February 2008 -- Pompe disease in children and adults: natural course, disease severity and impact on daily life |
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