RESEARCH 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.

  • January 2019
Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease

Human Gene Therapy
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  • December 2018
The ACE I/D polymorphism does not explain the heterogeneity of natural course and response to enzyme replacement therapy in Pompe disease

PLOS One
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  • November 2018
AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

Molecular Therapy: Methods and Clinical Developments
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  • November 2018
Satellite cells maintain regenerative capacity but fail to repair disease-associated muscle damage in mice with Pompe disease

Acta Neuropathologica Communications
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  • November 2018
Safety and efficacy of short- and long-term inspiratory muscle training in late-onset Pompe disease (LOPD): a pilot study

Journal of Neurology
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  • November 2018
Association of Muscle Strength and Walking Performance in Adult Patients With Pompe Disease

Physical Therapy
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  • October 2018
Clinical and molecular characterization of Korean children with infantile and late-onset Pompe disease: 10 years of experience with enzyme replacement therapy at a single center

Korean Journal of Pediatrics
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  • September 2018
Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease

Molecular Therapy
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  • August 2018
Pompe Disease: From Basic Science to Therapy

Neurotherapeutics
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  • August 2018
Pompe disease: how to solve many problems with one solution

Annals of Translational Medicine
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  • July 2018
Cardiac outcome in classic infantile Pompe disease after 13 years of treatment with recombinant human acid alpha-glucosidase

International Journal of Cardiology
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  • July 2018
MicroRNAs as Biomarkers in Pompe Disease

Genetics in Medicine
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  • July 2018
Severe Cardiac Involvement Is Rare in Patients with Late-Onset Pompe Disease and the Common c.-32-13T>G Variant: Implications for Newborn Screening

The Journal of Pediatrics: Clinical and Laboratory Observations
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  • June 2018
Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies

Stem Cell Reports
Abstract
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  • May 2018
Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients.

Orphanet Journal of Rare Diseases
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  • May 2018
Pompe disease treatment with twice a week high dose alglucoside alfa in a T patient with severe dilated cardiomyopathy

Molecular Genetics and Metabolism Reports
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  • April 2018
High Sustained Antibody Titers in Patients with Classic Infantile Pompe Disease Following Immunomodulation at Start of Enzyme Replacement Therapy

The Journal of Pediatrics
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  • April 2018
Therapeutic Benefit of Autophagy Modulation in Pompe Disease

Molecular Therapy
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  • March 2018
Perioperative management of children with glycogen storage disease type II-Pompe disease.

Pediatric Anesthesia
Abstract
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  • March 2018
Classic infantile Pompe patients approaching adulthood: a cohort study on consequences for the brain.

Developmental Medicine & Child Neurology
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  • March 2018
Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study

Genetics in Medicine
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  • March 2018
Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy

Journal of Inherited Metabolic Disease
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  • January 2018
Quantification of intramuscular fat in patients with late-onset Pompe disease by conventional magnetic resonance imaging for the long-term follow-up of enzyme replacement therapy

PLOS One
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  • December 2017
Immunomodulatory, liver depot gene therapy for Pompe disease

Cellular Immunology
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  • December 2017
Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation

Molecular Genetics and Metabolism
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  • December 2017
Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease

Human Gene Therapy Clinical Development
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  • November 2017
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

Science Translational Medicine
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  • November 2017
The humanistic burden of Pompe disease: are there still unmet needs? A systematic review

BMC Neurology
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  • November 2017
AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease

Nature: Scientific Reports
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  • September 2017
Atg5flox-Derived Autophagy Deficient Model of Pompe Disease: Does It Tell the Whole Story?

Molecular Therapy: Methods and Clinical Development
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  • August 2017
Sustained immune tolerance induction in enzyme replacement therapy–treated CRIM-negative patients with infantile Pompe disease

JCI Insight
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  • July 2017
A pilot study on using rapamycin-carrying synthetic vaccine particles (SVP) in conjunction with enzyme replacement therapy to induce immune tolerance in Pompe disease

Molecular Genetics and Metabolism Reports
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  • July 2017
Newborn Screening for Pompe Disease: Report from Pompe Disease Newborn Screening Working Group

Pediatrics
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  • July 2017
The Initial Evaluation of Patients After Positive Newborn Screening: Recommended Algorithms Leading to a Confirmed Diagnosis of Pompe Disease

Pediatrics
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  • July 2017
Management of Confirmed Newborn-Screened Patients With Pompe Disease Across the Disease Spectrum

Pediatrics
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  • June 2017
GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells

Molecular Therapy: Nucleic Acids
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  • May 2017
Skeletal muscle metabolism during prolonged exercise in Pompe disease.

Endocrine Connections
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  • April 2017
Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease

Molecular Genetics and Metabolism Reports
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  • March 2017
Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid a-Glucosidase in Pompe Patients Co-administered with Alglucosidase alpha

Molecular Therapy
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  • March 2017
Modulation of mTOR signaling as a strategy for the treatment of Pompe disease

EMBO Molecular Medicine
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  • March 2017
Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease.

Molecular Therapy: Nucleic Acids
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  • March 2017
Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction

Molecular Therapy: Methods and Clinical Development
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  • October 2016
Practical Recommendations for Diagnosis and Management of Respiratory Muscle Weakness in Late-Onset Pompe Disease

International Journal of Molecular Sciences
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  • February 2016
Very Early Treatment for Infantile-Onset Pompe Disease Contributes to Better Outcomes

The Journal of Pediatrics
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  • February 2015
Please click here to read all of the Abstracts and Poster Presentations from the Steps Forward meeting: Abstracts and Poster Presentations from the 7th European Symposium on Steps Forward in Pompe Disease: Turin, Italy, November 21–22, 2014 Below please find the Foreword from the Steps Forward Meeting.
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  • December 2014
Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease

Neuromuscular Disorders
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  • November 2014
Prevalence of Asymptomatic Vertebral Fractures in Late-Onset Pompe Disease

J Clin Endocrinol Metab
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  • September 2014
Identification and Characterization of Aberrant GAA Pre-mRNA Splicing in Pompe Disease Using a Generic Approach

Human Mutation
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  • May 2014
Adjunctive albuterol enhances the response to enzyme replacement therapy in late-onset Pompe disease

The FASEB Journal (May 2014) Courtesy of Sharon Krueger
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  • January 2014
The Nutrient-Responsive Transcription Factor TFE3 Promotes Autophagy, Lysosomal Biogenesis, and Clearance of Cellular Debris

Science Signaling
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  • January 2014
Increased aortic stiffness and blood pressure in non-classic Pompe disease

Journal of Inherited Metabolic Disorders
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  • January 2014
The value of muscle biopsies in Pompe disease: identifying lipofuscin inclusions in juvenile- and adult-onset patients

Acta Neuropathologica Communications
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  • November 2013
Phenotypical variation within 22 families with Pompe disease

Orphanet Journal of Rare Diseases
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  • October 2013
The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?

Orphanet Journal of Rare Diseases
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  • October 2013
Distinct disease phenotypes linked to different combinations of GAA mutations in a large late-onset GSDII sibship

Orphanet Journal of Rare Diseases
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  • October 2013
Early is better? A new algorithm for early diagnosis in Late Onset Pompe Disease (LOPD)

Acta Myologica
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  • August 2013
A Large-Scale Nationwide Newborn Screening Program for Pompe Disease in Taiwan: Towards Effective Diagnosis and Treatment

American Journal of Medical Genetics
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  • June 2013
Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material- Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT

PLOS ONE
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  • June 2013
Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy

Orphanet Journal of Rare Diseases
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  • June 2013
Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease

Journal of Neurology
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  • June 2013
Pain in adult patients with Pompe disease: A cross-sectional survey

Molecular Genetics and Metabolism
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  • June 2013
Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease

The American Society of Gene and Cell Therapy
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  • May 2013
A higher dose of enzyme therapy in patients with classic infantile Pompe disease seems to improve ventilator-free survival and motor function

BMC Musculoskeletal Disorders
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  • May 2013
Improvement of dysphagia in a child affected by Pompe disease treated with enzyme replacement therapy

Italian Journal of Pediatrics
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  • March 2013
Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study

Orphanet Journal of Rare Diseases
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  • March 2013
Enzyme replacement therapy and fatigue in adults with Pompe disease

Molecular Genetics and Metabolism
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  • March 2013
B-Cell Depletion and Immunomodulation before Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha-Glucosidase in Infantile-Onset Pompe Disease

Journal of Pediatrics
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  • February 2013
Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease

EMBO Molecular Medicine
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  • January 2013
Glycosylation-independent Lysosomal Targeting of Acid α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice

The Journal of Biological Chemistry
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  • February 2012
Autophagy and Mitochondria in Pompe Disease: Nothing is so New as What Has Long Been Forgotten

American Journal of Medical Genetics
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  • January 2012
Spinal Delivery of AAV Vector Restores Enzyme Activity and Increases Ventilation in Pompe Mice

The American Society of Gene and Cell Therapy
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  • November 2012
Case Report: Management of a Pregnancy Complicated by Pompe Disease

Obstetrics and Gynecology
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  • November 2012
Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study

Orphanet Journal of Rare Diseases
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  • October 2012
The Rasch-built Pompe-specific Activity (R-PAct) scale

Neuromuscular Disorders
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  • September 2012
Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study

Orphanet Journal of Rare Diseases
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  • September 2012
Severely impaired health status at diagnosis of Pompe disease: A cross-sectional analysis to explore the potential utility of neonatal screening

Molecular Genetics and Metabolism
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  • August 2012
Immune modulation in Pompe disease treated with enzyme replacement therapy

Expert Reviews in Clinical Immunology
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  • June 2012
Letter to the Editor: “Incontinence in Late-Onset Pompe Disease: An Underdiagnosed Treatable Condition”

European Neurology
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  • June 2012
A cross-sectional single-centre study on the spectrum of Pompe disease, German patients: molecular analysis of the GAA gene, manifestation and genotype-phenotype correlations.

Orphanet Journal of Rare Diseases
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  • June 2012
Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to an Adeno-Associated Virus Vector in Murine Pompe Disease

Biores Open Access
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  • December 2011
Genetic Implications of Newborn Screening

Acid Maltase Deficiency Association
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  • September 2011
Expanding the phenotype of late-onset pompe disease: Tongue weakness: A new clinical observation.

Wiley Online Library
Abstract
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  • September 2011
Facial-Muscle Weakness, Speech Disorders and Dysphagia are Common in Patients with Classic Infantile Pompe Disease Treated with Enzyme Therapy

Journal of Inherited Metabolic Disorders
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  • June 2011
Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

Orphanet Journal of Rare Diseases
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  • May 2011
Pompe disease gene therapy

Human Molecular Genetics
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  • May 2011
Suction-Modified Bergstrom Muscle Biopsy Technique: Experience with 13500 Procedures

Muscle and Nerve
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  • April 2011
Burden of illness of Pompe disease in patients only receiving supportive care

Journal of Inherited Metabolic Disease
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  • February 2011
Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle

Molecular Genetics and Metabolism
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  • December 2010
Where do we stand in enzyme replacement therapy in Pompe’s disease?

Neuromuscular Disorders
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  • November 2010
Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease

The Journal of Gene Medicine
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  • September 2010
Effect of enzyme therapy in juvenile patients with Pompe disease: A three-year open-label study

Neuromuscular Disorders
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  • August 2010
Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease: implications for therapy

Molecular Genetics and Metabolism
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  • August 2010
Hydrostatic Isolated Limb Perfusion with Adeno-associated Virus Vectors Enhances Correction of Skeletal Muscle in Pompe Disease

Gene Therapy
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  • August 2010
Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe disease

Molecular Genetics and Metabolism (Abstract Only)
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  • July 2010
Use of Cardiac Magnetic Resonance Imaging to Evaluate Cardiac Structure

Molecular Genetics Metabolism
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  • July 2010
Hearing loss in Pompe disease revisited: results from a study of 24 children

Journal of Inherited Metabolic Disease
Abstract
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  • July 2010
Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype

Blood (Abstract Only)
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  • June 2010
Low bone mass in Pompe disease: Muscular strength as a predictor of bone mineral density

Bone
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  • June 2010
Termination of autophagy and reformation of lysosomes regulated by mTOR

Nature
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  • April 2010
A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe’s Disease

The NEW ENGLAND JOURNAL of MEDICINE
Abstract
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  • April 2010
The angiotensin-converting enzyme insertion/deletion polymorphism modifies the clinical outcome in patients with Pompe disease

Genetics in Medicine, Courtesy of Monty Frost
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  • January 2010
Therapy for Lysosomal Storage Disorders

Life
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  • December 2009
Immunomodulatory Gene Therapy in Lysosomal Storage Disorders

Current Gene Therapy
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  • November 2009
Desensitization of an adult patient with Pompe disease and a history of anaphylaxis to alglucosidase alfa

Molecular Genetics and Metabolism (Abstract Only)
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  • November 2009
Fatigue in neuromuscular disorders: Focus on Guillain-Barré syndrome and Pompe disease

Cellular and Molecular Life Sciences
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  • October 2009
Therapeutic approaches in Glycogen Storage Disease type II (GSDII)/Pompe disease

Neurotherapeutics
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  • September 2009
Early Treatment With Alglucosidase Alfa Prolongs Long-Term Survival of Infants With Pompe Disease

Pediatric Research
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  • August 2009
Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

Molecular Genetics and Metabolism
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  • August 2009
Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease

Molecular Therapy
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  • July 2009
The values and limits of an in vitro model of Pompe disease: The best laid schemes o’ mice an’ men.

Autophagy
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  • June 2009
Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease

Molecular Therapy
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  • June 2009
The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts

Molecular Therapy
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  • April 2009
Murine Muscle Cell Models for Pompe Disease and Their Use in Studying Therapeutic Approaches

Molecular Genetics and Metabolism
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  • April 2009
Side-Alternating Vibration Training Improves Muscle Performance in a Patient with Late-Onset Pompe Disease

Case Reports in Medicine
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  • February 2009
Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease

Neuromuscular Disorders (Abstract Only)
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  • September 2008
A Dutch guideline for the treatment of scoliosis in neuromuscular disorders

Scoliosis
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  • June 2008
Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus–mediated Gene Therapy

Molecular Therapy
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  • June 2008
Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease

Neuromuscular Disorders (Abstract Only)
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  • December 2007
Deconstructing Pompe Disease by Analyzing Single Muscle Fibers

Autophagy
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  • November 2007
Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune Tolerance

The American Journal of Human Genetics
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  • January 2007
Fractures in children with Pompe disease: a potential long-term complication

Pediatric Radiology
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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 improve transport and enhance the stability of mutant a-glucosidases in glycogen storage disease type II

Molecular Genetics and Metabolism, September 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
Abstract
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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
Abstract
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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
Abstract
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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
Abstract
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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
Abstract
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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)
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 Acid Maltase Deficiency Association

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Your generous contribution will go a long way into improving the quality of life of Pompe Disease Patients worldwide. Donations go towards Pompe Disease Research.

If you are interested in learning more about Pompe Disease and would like to make a contribution in support of necessary research, please contact us at:

(210) 494-6144

info@amda-pompe.org

AMDA

PO Box 700248

San Antonio, Texas 78270 USA