-
6
-
-
0020009878
-
Gaucher disease of the Norrbottnian type (type III). Phenotypic manifestations
-
(1982)
Prog Clin Biol Res
, vol.95
, pp. 67-94
-
-
Svennerholm, L.1
Dreborg, S.2
Erikson, A.3
Groth, C.G.4
Hillborg, P.O.5
Hakansson, G.6
Nilsson, O.7
Tibblin, E.8
-
8
-
-
0001775305
-
A new familial, infantile form of diffuse brain sclerosis
-
(1916)
Brain
, vol.39
, pp. 74-114
-
-
Krabbe, K.1
-
12
-
-
0032537062
-
Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy
-
(1998)
N Engl J Med
, vol.338
, pp. 1119-1126
-
-
Krivit, W.1
Shapiro, E.G.2
Peters, C.3
Wagner, J.E.4
Comu, G.5
Kurtzberg, J.6
Wenger, D.A.7
Kolodny, E.H.8
Vanier, M.T.9
Loes, D.J.10
Dusenbery, K.11
Lockman, L.A.12
-
15
-
-
0032947981
-
Molecular heterogeneity of Krabbe disease
-
(1999)
J Inherit Metab Dis
, vol.22
, pp. 155-162
-
-
Fu, L.1
Inui, K.2
Nishigaki, T.3
Tatsumi, N.4
Tsukamoto, H.5
Kokubu, C.6
Muramatsu, T.7
Okada, S.8
-
18
-
-
0033882996
-
Distribution and characterization of GFP(+) donor hematogenous cells in Twitcher mice after bone marrow transplantation
-
(2000)
Am J Pathol
, vol.156
, pp. 1849-1854
-
-
Wu, Y.P.1
McMahon, E.2
Kraine, M.R.3
Tisch, R.4
Meyers, A.5
Frelinger, J.6
Matsushima, G.K.7
Suzuki, K.8
-
20
-
-
13344286310
-
Molecular cloning and expression of cDNA for murine galactocerebrosidase and mutation analysis of the twitcher mouse, a model of Krabbe's disease
-
(1996)
J Neurochem
, vol.66
, pp. 1118-1124
-
-
Sakai, N.1
Inui, K.2
Tatsumi, N.3
Fukushima, H.4
Nishigaki, T.5
Taniike, M.6
Nishimoto, J.7
Tsukamoto, H.8
Yanagihara, I.9
Ozono, K.10
Okada, S.11
-
23
-
-
0029084244
-
The twitcher mouse: A model for Krabbe disease and for experimental therapies
-
(1995)
Brain Pathol
, vol.5
, pp. 249-258
-
-
Suzuki, K.1
-
31
-
-
0034515314
-
Treating glucosphingolipid disorders by chemotherapy: Use of approved drugs and over-the-counter remedies
-
(2000)
J Inherit Metab Dis
, vol.23
, pp. 767-777
-
-
Radin, N.S.1
-
32
-
-
0033060380
-
Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin
-
(1999)
Proc Natl Acad Sci USA
, vol.96
, pp. 6388-6393
-
-
Jeyakumar, M.1
Butters, T.D.2
Cortina-Borja, M.3
Hunnam, V.4
Proia, R.L.5
Perry, V.H.6
Dwek, R.A.7
Platt, F.M.8
-
34
-
-
0034728914
-
Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis
-
(2000)
Lancet
, vol.355
, pp. 1481-1485
-
-
Cox, T.1
Lachmann, R.2
Hollak, C.3
Aerts, J.4
Van Weely, S.5
Hrebicek, M.6
Platt, F.7
Butters, T.8
Dwek, R.9
Moyses, C.10
Gow, I.11
Elstein, D.12
Zimran, A.13
-
41
-
-
0035168688
-
Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation
-
(2001)
Blood
, vol.97
, pp. 327-329
-
-
Jeyakumar, M.1
Norflus, F.2
Tifft, C.J.3
Cortina-Borja, M.4
Butters, T.D.5
Proia, R.L.6
Perry, V.H.7
Dwek, R.A.8
Platt, F.M.9
-
49
-
-
0016147017
-
The turnover of myelin phosphatidylcholine and sphingomyelin in the adult rat brain
-
(1974)
Brain Res
, vol.78
, pp. 99-108
-
-
Jungalwala, F.B.1
-
52
-
-
0032941197
-
Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and Sly syndromes, and Gaucher disease type III
-
(1999)
Curr Opin Neurol
, vol.12
, pp. 167-176
-
-
Krivit, W.1
Peters, C.2
Shapiro, E.G.3
|