Pompe Disease: Long-Term Follow-up Clinical Guidelines
Pompe disease treatment with twice a week high dose alglucoside alfa in a patient with severe dilated cardiomyopathy - ScienceDirect
![PDF] The Initial Evaluation of Patients After Positive Newborn Screening: Recommended Algorithms Leading to a Confirmed Diagnosis of Pompe Disease | Semantic Scholar](https://d3i71xaburhd42.cloudfront.net/70a69e34463c8a68d377184d96bfbeb9ffcf5a96/3-Figure1-1.png "PDF] The Initial Evaluation of Patients After Positive Newborn Screening: Recommended Algorithms Leading to a Confirmed Diagnosis of Pompe Disease | Semantic Scholar")
PDF] The Initial Evaluation of Patients After Positive Newborn Screening: Recommended Algorithms Leading to a Confirmed Diagnosis of Pompe Disease | Semantic Scholar
Pompe Disease: a Clinical, Diagnostic, and Therapeutic Overview | Current Treatment Options in Neurology
IJNS | Free Full-Text | Newborn Screening for Pompe Disease: Pennsylvania Experience
JCI Insight - Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses
SEC Filing | Amicus Therapeutics
Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease
Immunomodulatory, liver depot gene therapy for Pompe disease - ScienceDirect
Lessons Learned from Pompe Disease Newborn Screening and Follow-up - Document - Gale Academic OneFile
Oligosaccharide concentration in urine from controls and Pompe... | Download Table
Glucose tetrasaccharide as a biomarker for monitoring the therapeutic response to enzyme replacement therapy for Pompe disease - ScienceDirect
Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease: Molecular Therapy - Methods & Clinical Development
Glucose tetrasaccharide as a biomarker for monitoring the therapeutic response to enzyme replacement therapy for Pompe disease - ScienceDirect
Course of the urinary Hex4 index patient. | Download Scientific Diagram
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Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy | Orphanet Journal of Rare Diseases | Full Text
SEC Filing | Amicus Therapeutics
Topeak Yhex Speed Wrench Hex 4/5/6mm | Mammoth
Children's Hospital of Philadelphia Glycomics Profile Analysis by MALDI TOF/MS in Human CSF Xueli Li, Ph.D., Children's Hospital of Philadelphia. - ppt download
Case study: monitoring of Glc4 tetrasaccharide in the urine of Pompe patients, use of MALDI-TOF MS, and 1H NMR | SpringerLink
The new era of Pompe disease: Advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management - Kishnani - 2012 - American Journal of Medical Genetics Part C: Seminars in
Case study: monitoring of Glc4 tetrasaccharide in the urine of Pompe patients, use of MALDI-TOF MS, and 1H NMR | SpringerLink
SEC Filing | Amicus Therapeutics
