Punita Gupta Brian J Shayota Ankit K Desai Fuad Kiblawi Dorothy Myridakis John Messina Peter Tah Lorien Tambini-King Priya S Kishnani
10.3389/fimmu.2020.01929
Front Immunol
2020 Sep 4;11:1929.
crim negative anti-drug antibodies early diagnosis immunomodulation infantile pompe disease newborn screening
CC BY
https://pubmed.ncbi.nlm.nih.gov/33013846/
Early Medical Intervention Enzyme Replacement Therapy Glycogen Storage Disease Type II / therapy Immune Tolerance / drug effects Immunosuppressive Agents / administration & dosage alpha-Glucosidases / therapeutic use
Early Medical Intervention Enzyme Replacement Therapy Glycogen Storage Disease Type II / therapy Immune Tolerance / drug effects Immunosuppressive Agents / administration & dosage alpha-Glucosidases / therapeutic use Case Reports Antibodies / blood Female Genetic Predisposition to Disease Genetic Testing Glycogen Storage Disease Type II / diagnosis Glycogen Storage Disease Type II / genetics Glycogen Storage Disease Type II / immunology Humans Infant, Newborn Mutation Phenotype Prenatal Diagnosis Treatment Outcome alpha-Glucosidases / genetics alpha-Glucosidases / immunology Antibodies Immunosuppressive Agents GAA protein, human alpha-Glucosidases
PMC7498628
33013846
A Race Against Time-Changing the Natural History of CRIM Negative Infantile Pompe Disease
2020