Evidence that l-Carnitine and Selenium Supplementation Reduces Oxidative Stress in Phenylketonuric Patients
Sitta, A., Vanzin, C. S., Biancini, G. B., Manfredini, V., de Oliveira, A. B., Wayhs, C. A. Y., Ribas, G. O. S., Giugliani, L., Schwartz, I. V. D., Bohrer, D., Garcia, S. C., Wajner, M., Vargas, C. R.
Published in Cellular and molecular neurobiology (01.04.2011)
Published in Cellular and molecular neurobiology (01.04.2011)
Get full text
Journal Article
In vitro effect of genistein on DNA damage in leukocytes from mucopolysaccharidosis IVA patients
Negretto, G.W., Deon, M., Burin, M., Biancini, G.B., Ribas, G., Garcia, S.C., Goethel, G., Fracasso, R., Giugliani, L., Giugliani, R., Vargas, C.R.
Published in Molecular genetics and metabolism (01.02.2014)
Published in Molecular genetics and metabolism (01.02.2014)
Get full text
Journal Article
13 - ELADOCAGENE EXUPARVOVEC GENE THERAPY IMPROVES MOTOR DEVELOPMENT IN PATIENTS WITH AROMATIC L-AMINO ACID DECARBOXYLASE DEFICIENCY
Pachelli, P.E., Giugliani, L., Federhen, A., Wang, A., Russel, A., Roubertie, A., Chien, Y-H., Lee, N-C., Tai, C-H., Wuh-Liang, P.
Published in Cytotherapy (Oxford, England) (01.10.2022)
Published in Cytotherapy (Oxford, England) (01.10.2022)
Get full text
Journal Article
ELADOCAGENE EXUPARVOVEC GENE THERAPY IMPROVES MOTOR DEVELOPMENT IN PATIENTS WITH AROMATIC L-AMINO ACID DECARBOXYLASE DEFICIENCY
Pachelli, P.E., Giugliani, L., Federhen, A., Wang, A., Russel, A., Roubertie, A., Chien, Y-H., Lee, N-C., Tai, C-H., Wuh-Liang, P.
Published in Cytotherapy (Oxford, England) (01.10.2022)
Published in Cytotherapy (Oxford, England) (01.10.2022)
Get full text
Journal Article
Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study
Patterson, Marc C, Mengel, Eugen, Vanier, Marie T, Schwierin, Barbara, Muller, Audrey, Cornelisse, Peter, Pineda, Mercè
Published in Orphanet journal of rare diseases (28.05.2015)
Published in Orphanet journal of rare diseases (28.05.2015)
Get full text
Journal Article
SAFETY AND IMPROVED EFFICACY OUTCOMES IN CHILDREN WITH AADC DEFICIENCY TREATED WITH ELADOCAGENE EXUPARVOVEC GENE THERAPY: RESULTS FROM THREE CLINICAL TRIALS
Hwu, PWL, Pachelli, PE, Chien, YH, Lee, NC, Tseng, SH, Tai, CH, Conway, AM, Federhen, A, Giugliani, L, Felix, T, Pykett, M
Published in Cytotherapy (Oxford, England) (01.04.2021)
Published in Cytotherapy (Oxford, England) (01.04.2021)
Get full text
Journal Article