-
3
-
-
33747209013
-
A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)
-
Muenzer J., Wraith J.E., Beck M., Giugliani R., Harmatz P., Eng C.M., Vellodi A., Martin R., Ramaswami U., Gucsavas-Calikoglu M., et al. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med 8 8 (2006) 465-473
-
(2006)
Genet Med
, vol.8
, Issue.8
, pp. 465-473
-
-
Muenzer, J.1
Wraith, J.E.2
Beck, M.3
Giugliani, R.4
Harmatz, P.5
Eng, C.M.6
Vellodi, A.7
Martin, R.8
Ramaswami, U.9
Gucsavas-Calikoglu, M.10
-
4
-
-
0025869216
-
Replacement therapy for inherited enzyme deficiency-macrophage-targeted glucocerobrosidase for Gaucher's disease
-
Barton N.W., Brady R.O., Dambrosia J.M., Di Bisceglie A.M., Doppelt S.H., Hill S.C., Mankin H.J., Murria G.J., Parker R.I., Argoff C.E., et al. Replacement therapy for inherited enzyme deficiency-macrophage-targeted glucocerobrosidase for Gaucher's disease. New Eng J Med 324 (1991) 1464-1470
-
(1991)
New Eng J Med
, vol.324
, pp. 1464-1470
-
-
Barton, N.W.1
Brady, R.O.2
Dambrosia, J.M.3
Di Bisceglie, A.M.4
Doppelt, S.H.5
Hill, S.C.6
Mankin, H.J.7
Murria, G.J.8
Parker, R.I.9
Argoff, C.E.10
-
5
-
-
0035816007
-
Enzyme replacement therapy in Fabry disease: a randomized controlled trial
-
Schiffmann R., Kopp J.B., Austin III H.A., Sabnis S., Moore D.F., Weibel T., Balow J.E., and Brady R.O. Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA 285 (2001) 2743-2749
-
(2001)
JAMA
, vol.285
, pp. 2743-2749
-
-
Schiffmann, R.1
Kopp, J.B.2
Austin III, H.A.3
Sabnis, S.4
Moore, D.F.5
Weibel, T.6
Balow, J.E.7
Brady, R.O.8
-
6
-
-
0035811624
-
Safety and efficacy of recombinant human alphagalactosidase A replacement in Fabry's disease
-
Eng C.M., Guffon N., Wilcox W.R., Germain D.P., Lee P., Waldek S., Caplan L., Linthorst G.E., and Desnick R.J. Safety and efficacy of recombinant human alphagalactosidase A replacement in Fabry's disease. New Eng J Med 345 (2001) 9-16
-
(2001)
New Eng J Med
, vol.345
, pp. 9-16
-
-
Eng, C.M.1
Guffon, N.2
Wilcox, W.R.3
Germain, D.P.4
Lee, P.5
Waldek, S.6
Caplan, L.7
Linthorst, G.E.8
Desnick, R.J.9
-
7
-
-
2342666229
-
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo controlled, multinational study of recombinant human α-l-iduronidase (laronidase)
-
Wraith J.E., Clarke L.A., Beck M., Kolodny E.H., Pastores G.M., Muenzaer J., Rapoport D.M., Berger K.I., Swiedler S.J., Kakkis E.D., et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo controlled, multinational study of recombinant human α-l-iduronidase (laronidase). J Pediatrics 144 (2004) 581-588
-
(2004)
J Pediatrics
, vol.144
, pp. 581-588
-
-
Wraith, J.E.1
Clarke, L.A.2
Beck, M.3
Kolodny, E.H.4
Pastores, G.M.5
Muenzaer, J.6
Rapoport, D.M.7
Berger, K.I.8
Swiedler, S.J.9
Kakkis, E.D.10
-
8
-
-
0035905889
-
Enzymereplacement therapy in mucopolysaccharidosis I
-
Kakis E.D., Muenzer J., Tiller G.E., Waber L., Belmont J., Passage M., Izykowski B., Phillips J., Doroshow R., Wallot I., Hoft R., and Neufeld E.F. Enzymereplacement therapy in mucopolysaccharidosis I. New Eng J Med 344 (2001) 182-188
-
(2001)
New Eng J Med
, vol.344
, pp. 182-188
-
-
Kakis, E.D.1
Muenzer, J.2
Tiller, G.E.3
Waber, L.4
Belmont, J.5
Passage, M.6
Izykowski, B.7
Phillips, J.8
Doroshow, R.9
Wallot, I.10
Hoft, R.11
Neufeld, E.F.12
-
9
-
-
27744493202
-
Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a Phase 2 open-label clinical study of recombinant human Nacetylgalactosidase-4-sulfatase
-
Harmatz P., Ketteridge D., Giugliani R., Guffon N., Teles E.L., Miranda M.C., Yu Z.F., Swiedler S.J., and Hopwood J.J. Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a Phase 2 open-label clinical study of recombinant human Nacetylgalactosidase-4-sulfatase. Pediatrics 115 (2005) e681-e689
-
(2005)
Pediatrics
, vol.115
-
-
Harmatz, P.1
Ketteridge, D.2
Giugliani, R.3
Guffon, N.4
Teles, E.L.5
Miranda, M.C.6
Yu, Z.F.7
Swiedler, S.J.8
Hopwood, J.J.9
|