Fabry disease in the Spanish population: observational study with detection of 77 patients.
Orphanet Journal Of Rare Diseases
Vieitez, Irene I; Souto-Rodriguez, Olga O; Fernandez-Mosquera, Lorena L; San Millan, Beatriz B; Teijeira, Susana S; Fernandez-Martin, Julian J; Martinez-Sanchez, Felisa F; Aldamiz-Echevarria, Luis Jose LJ; Lopez-Rodriguez, Monica M; Navarro, Carmen C; Ortolano, Saida S
Co-administration with the pharmacological chaperone AT1001 increases recombinant human α-galactosidase A tissue uptake and improves substrate reduction in Fabry mice.
Molecular Therapy : The Journal Of The American Society Of Gene Therapy
Benjamin, Elfrida R ER; Khanna, Richie R; Schilling, Adriane A; Flanagan, John J JJ; Pellegrino, Lee J LJ; Brignol, Nastry N; Lun, Yi Y; Guillen, Darlene D; Ranes, Brian E BE; Frascella, Michelle M; Soska, Rebecca R; Feng, Jessie J; Dungan, Leo L; Young, Brandy B; Lockhart, David J DJ; Valenzano, Kenneth J KJ