Characterization of immune response in Cross-Reactive Immunological Material (CRIM)-positive infantile Pompe disease patients treated with enzyme replacement therapy.
Mol Genet Metab Rep
; 20: 100475, 2019 Sep.
Article
en En
| MEDLINE
| ID: mdl-31193175
AIMS, Alberta infant motor scale; Anti-rhGAA Ig antibodies; Antidrug antibodies; CI-MPR, Cation-independent mannose 6-phosphate receptor; CRIM, Cross-reactive immunological material; EOW, Every other week; ERT, Enzyme replacement therapy; Enzyme replacement therapy; GAA, Acid α-glucosidase; GAA, Gene encoding acid α-glucosidase; Glc4, Glucose tetrasaccharide; Glycogen storage disease type II; HLA, Human leukocyte antigen; HSAT, High and sustained antibody titers; IPD, Infantile Pompe disease; IgG, Immunoglobulin G; LT, Low titers; LVMI, Left ventricular mass index; MHC, Major histocompatibility complex; Neuromuscular disease; Pompe disease; RUSP, Recommended universal screening panel; SIT, Sustained intermediate titers; iTEM, Individualized T-cell epitope measure; rhGAA, Recombinant human acid α-glucosidase
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Tipo de estudio:
Prognostic_studies
Idioma:
En
Revista:
Mol Genet Metab Rep
Año:
2019
Tipo del documento:
Article
País de afiliación:
Estados Unidos
