IJNS | Free Full-Text | Newborn Screening for Pompe Disease in Illinois: Experience with 684,290 Infants
IJNS | Free Full-Text | Newborn Screening for Pompe Disease: Pennsylvania Experience
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Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease
SEC Filing | Amicus Therapeutics
Long-term monitoring of patients with infantile-onset Pompe disease on enzyme replacement therapy using a urinary glucose tetrasaccharide biomarker | Genetics in Medicine
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Glucose tetrasaccharide as a biomarker for monitoring the therapeutic response to enzyme replacement therapy for Pompe disease - ScienceDirect
SEC Filing : AVROBIO
Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease
Hex4 levels. Diagnosis and onset of IAR are indicated by black and... | Download Scientific Diagram
Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy | Orphanet Journal of Rare Diseases | Full Text
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Course of the urinary Hex4 index patient. | Download Scientific Diagram
Immunomodulatory, liver depot gene therapy for Pompe disease - ScienceDirect
IJNS | Free Full-Text | Newborn Screening for Pompe Disease in Illinois: Experience with 684,290 Infants
Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease: Molecular Therapy - Methods & Clinical Development
Pompe Disease: a Clinical, Diagnostic, and Therapeutic Overview | Current Treatment Options in Neurology
IJNS | Free Full-Text | Newborn Screening for Pompe Disease: Pennsylvania Experience
SEC Filing | Amicus Therapeutics
SEC Filing | Amicus Therapeutics
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