-
1
-
-
49649085254
-
Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1
-
19047232 10.1542/peds.2007-2144
-
Andersson H, Kaplan P, Kacena K, Yee J (2008) Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1. Pediatrics 122:1182-1190
-
(2008)
Pediatrics
, vol.122
, pp. 1182-1190
-
-
Andersson, H.1
Kaplan, P.2
Kacena, K.3
Yee, J.4
-
2
-
-
0036073124
-
Bone complications in children with Gaucher disease
-
12036831
-
Bembi B, Ciana G, Mengel E, Terk MR, Martini C, Wenstrup RJ (2002) Bone complications in children with Gaucher disease. Br J Radiol 75(Suppl 1):A37-A44
-
(2002)
Br J Radiol
, vol.75
, Issue.SUPPL. 1
-
-
Bembi, B.1
Ciana, G.2
Mengel, E.3
Terk, M.R.4
Martini, C.5
Wenstrup, R.J.6
-
4
-
-
0034626360
-
The Gaucher registry: Demographics and disease characteristics of 1698 patients with Gaucher disease
-
11025794 10.1001/archinte.160.18.2835 1:STN:280:DC%2BD3cvnvVelsQ%3D%3D
-
Charrow J, Andersson HC, Kaplan P, Kolodny EH, Mistry P, Pastores G, Rosenbloom BE, Scott CR, Wappner RS, Weinreb NJ, Zimran A (2000) The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease. Arch Intern Med 160:2835-2843
-
(2000)
Arch Intern Med
, vol.160
, pp. 2835-2843
-
-
Charrow, J.1
Andersson, H.C.2
Kaplan, P.3
Kolodny, E.H.4
Mistry, P.5
Pastores, G.6
Rosenbloom, B.E.7
Scott, C.R.8
Wappner, R.S.9
Weinreb, N.J.10
Zimran, A.11
-
5
-
-
25144469366
-
Gaucher disease and bone: Laboratory and skeletal mineral density variations during a long period of enzyme replacement therapy
-
16151904 10.1007/s10545-005-0032-y 1:CAS:528:DC%2BD2MXpvVWqu74%3D
-
Ciana G, Addobbati R, Tamaro G, Leopaldi A, Nevyjel M, Ronfani L, Vidoni L, Pittis MG, Bembi B (2005) Gaucher disease and bone: laboratory and skeletal mineral density variations during a long period of enzyme replacement therapy. J Inherit Metab Dis 28:723-732
-
(2005)
J Inherit Metab Dis
, vol.28
, pp. 723-732
-
-
Ciana, G.1
Addobbati, R.2
Tamaro, G.3
Leopaldi, A.4
Nevyjel, M.5
Ronfani, L.6
Vidoni, L.7
Pittis, M.G.8
Bembi, B.9
-
6
-
-
78651236390
-
Osseous manifestations of adult Gaucher disease in the era of enzyme replacement therapy
-
21200186 10.1097/MD.0b013e3182057be4 1:CAS:528:DC%2BC3MXhs1Ohtg%3D%3D
-
Deegan PB, Pavlova E, Tindall J, Stein PE, Bearcroft P, Mehta A, Hughes D, Wraith JE, Cox TM (2011) Osseous manifestations of adult Gaucher disease in the era of enzyme replacement therapy. Medicine 90:52-60
-
(2011)
Medicine
, vol.90
, pp. 52-60
-
-
Deegan, P.B.1
Pavlova, E.2
Tindall, J.3
Stein, P.E.4
Bearcroft, P.5
Mehta, A.6
Hughes, D.7
Wraith, J.E.8
Cox, T.M.9
-
7
-
-
33645681688
-
Enzyme replacement therapy and bony changes in Egyptian paediatric Gaucher disease patients
-
10.1007/s10545-006-0121-6 1:CAS:528:DC%2BD28XjtlWgtbc%3D
-
El-Beshlawy A, Ragab L, Youssry I, Yakout K, El-Kiki H, Eid K, Mansour IM, Abd El-Hamid S, Yang M, Mistry PK (2006) Enzyme replacement therapy and bony changes in Egyptian paediatric Gaucher disease patients. Inherit Metab Dis 29:92-98
-
(2006)
Inherit Metab Dis
, vol.29
, pp. 92-98
-
-
El-Beshlawy, A.1
Ragab, L.2
Youssry, I.3
Yakout, K.4
El-Kiki, H.5
Eid, K.6
Mansour, I.M.7
Abd El-Hamid, S.8
Yang, M.9
Mistry, P.K.10
-
8
-
-
39549119956
-
Gaucher disease: Phenotypic and genetic variation, Chapter 146.1
-
C.R. Scriver A.L. Beaudet W.S. Sly D. Valle (eds) 9 McGraw-Hill New York
-
Grabowski GA, Kolodny EH, Weinreb NJ, Charrow J, Pastores GM, Mistry PK (2006) Gaucher disease: phenotypic and genetic variation, Chapter 146.1. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds) The metabolic and molecular bases of inherited disease, 9th edn. McGraw-Hill, New York
-
(2006)
The Metabolic and Molecular Bases of Inherited Disease
-
-
Grabowski, G.A.1
Kolodny, E.H.2
Weinreb, N.J.3
Charrow, J.4
Pastores, G.M.5
Mistry, P.K.6
-
9
-
-
0029846221
-
Acceleration of retarded growth in children with Gaucher disease after treatment with alglucerase
-
8757576 10.1016/S0022-3476(96)70203-2 1:CAS:528:DyaK28XltF2iurw%3D
-
Kaplan P, Mazur A, Manor O, Charrow J, Esplin J, Gribble TJ, Wappner RS, Wisch JS, Weinreb NJ (1996) Acceleration of retarded growth in children with Gaucher disease after treatment with alglucerase. J Pediatr 129:149-153
-
(1996)
J Pediatr
, vol.129
, pp. 149-153
-
-
Kaplan, P.1
Mazur, A.2
Manor, O.3
Charrow, J.4
Esplin, J.5
Gribble, T.J.6
Wappner, R.S.7
Wisch, J.S.8
Weinreb, N.J.9
-
10
-
-
33751561194
-
Pediatric BMD reference database for US white children
-
Kelly TL, Specker BL, Binkely T, Zemel BS, Leonard MB, Kalkwarf HJ, Moyer-Mileur LJ, Shepard JA (2005) Pediatric BMD reference database for US white children. Bone 36:S30
-
(2005)
Bone
, vol.36
, pp. 30
-
-
Kelly, T.L.1
Specker, B.L.2
Binkely, T.3
Zemel, B.S.4
Leonard, M.B.5
Kalkwarf, H.J.6
Moyer-Mileur, L.J.7
Shepard, J.A.8
-
11
-
-
51249109271
-
Special report on the 2007 adult and pediatric Position Development Conferences of the International Society for Clinical Densitometry
-
18633664 10.1007/s00198-008-0689-9 1:STN:280:DC%2BD1crnvV2hsg%3D%3D
-
Lewiecki EM, Gordon CM, Baim S, Binkley N, Bilezikian JP, Kendler DL, Hans DB, Silverman S, Bishop NJ, Leonard MB, Bianchi ML, Kalkwarf HJ, Langman CB, Plotkin H, Rauch F, Zemel BS (2008) Special report on the 2007 Adult and Pediatric Position Development Conferences of the International Society for Clinical Densitometry. Osteoporos Int 19:1369-1378
-
(2008)
Osteoporos Int
, vol.19
, pp. 1369-1378
-
-
Lewiecki, E.M.1
Gordon, C.M.2
Baim, S.3
Binkley, N.4
Bilezikian, J.P.5
Kendler, D.L.6
Hans, D.B.7
Silverman, S.8
Bishop, N.J.9
Leonard, M.B.10
Bianchi, M.L.11
Kalkwarf, H.J.12
Langman, C.B.13
Plotkin, H.14
Rauch, F.15
Zemel, B.S.16
-
12
-
-
38549096540
-
Recommendations for the assessment and monitoring of skeletal manifestations in children with Gaucher disease
-
18094966 10.1007/s00256-007-0425-0 1:STN:280:DC%2BD1c%2FmtFKjtw%3D%3D
-
Maas M, Hangartner T, Mariani G, McHugh K, Moore S, Grabowski GA, Kaplan P, Vellodi A, Yee J, Steinbach L (2008) Recommendations for the assessment and monitoring of skeletal manifestations in children with Gaucher disease. Skeletal Radiol 37:185-188
-
(2008)
Skeletal Radiol
, vol.37
, pp. 185-188
-
-
Maas, M.1
Hangartner, T.2
Mariani, G.3
McHugh, K.4
Moore, S.5
Grabowski, G.A.6
Kaplan, P.7
Vellodi, A.8
Yee, J.9
Steinbach, L.10
-
13
-
-
78650827299
-
Osteopenia in Gaucher disease develops early in life: Response to imiglucerase enzyme therapy in children, adolescent and adults
-
21112800 10.1016/j.bcmd.2010.10.011
-
Mistry PK, Weinreb NJ, Kaplan P, Cole JA, Gwosdow AR, Hangartner T (2011) Osteopenia in Gaucher disease develops early in life: response to imiglucerase enzyme therapy in children, adolescent and adults. Blood Cells Mol Dis 46:66-72
-
(2011)
Blood Cells Mol Dis
, vol.46
, pp. 66-72
-
-
Mistry, P.K.1
Weinreb, N.J.2
Kaplan, P.3
Cole, J.A.4
Gwosdow, A.R.5
Hangartner, T.6
-
15
-
-
0036308134
-
Correlation of bone marrow response with hematological, biochemical, and visceral responses to enzyme replacement therapy of nonneuronopathic (type 1) Gaucher disease in 30 adult patients
-
12064917 10.1006/bcmd.2002.0511
-
Poll LW, Koch JA, Willers R, Aerts H, Scherer A, Häussinger D, Mödder U, vom Dahl S (2002) Correlation of bone marrow response with hematological, biochemical, and visceral responses to enzyme replacement therapy of nonneuronopathic (type 1) Gaucher disease in 30 adult patients. Blood Cells Mol Dis 28:209-220
-
(2002)
Blood Cells Mol Dis
, vol.28
, pp. 209-220
-
-
Poll, L.W.1
Koch, J.A.2
Willers, R.3
Aerts, H.4
Scherer, A.5
Häussinger, D.6
Mödder, U.7
Vom Dahl, S.8
-
16
-
-
0029155493
-
Enzyme replacement therapy for Gaucher disease: Skeletal responses to macrophage-targeted glucocerebrosidase
-
7567322 1:STN:280:DyaK28%2FgtVWmsg%3D%3D
-
Rosenthal DI, Doppelt SH, Mankin HJ, Dambrosia JM, Xavier RJ, McKusick KA, Rosen BR, Baker J, Niklason LT, Hill SC, et al.(1995) Enzyme replacement therapy for Gaucher disease: skeletal responses to macrophage-targeted glucocerebrosidase. Pediatrics 96:629-637
-
(1995)
Pediatrics
, vol.96
, pp. 629-637
-
-
Rosenthal, D.I.1
Doppelt, S.H.2
Mankin, H.J.3
Dambrosia, J.M.4
Xavier, R.J.5
McKusick, K.A.6
Rosen, B.R.7
Baker, J.8
Niklason, L.T.9
Hill, S.C.10
-
17
-
-
42049119012
-
Improvement of bone disease by imiglucerase (Cerezyme) therapy in patients with skeletal manifestations of type 1 Gaucher disease: Results of a 48-month longitudinal cohort study
-
18312448 10.1111/j.1399-0004.2008.00978.x 1:STN:280: DC%2BD1c3jvFCmsw%3D%3D
-
Sims KB, Pastores GM, Weinreb NJ, Barranger J, Rosenbloom BE, Packman S, Kaplan P, Mankin H, Xavier R, Angell J, Fitzpatrick MA, Rosenthal D (2008) Improvement of bone disease by imiglucerase (Cerezyme) therapy in patients with skeletal manifestations of type 1 Gaucher disease: results of a 48-month longitudinal cohort study. Clin Genet 73:430-440
-
(2008)
Clin Genet
, vol.73
, pp. 430-440
-
-
Sims, K.B.1
Pastores, G.M.2
Weinreb, N.J.3
Barranger, J.4
Rosenbloom, B.E.5
Packman, S.6
Kaplan, P.7
Mankin, H.8
Xavier, R.9
Angell, J.10
Fitzpatrick, M.A.11
Rosenthal, D.12
-
18
-
-
0022410240
-
Skeletal complications of Gaucher disease
-
4033409 10.1097/00005792-198509000-00003 1:STN:280:DyaL2M3psFeksQ%3D%3D
-
Stowens DW, Teitelbaum SL, Kahn AJ, Barranger JA (1985) Skeletal complications of Gaucher disease. Medicine 64:310-322
-
(1985)
Medicine
, vol.64
, pp. 310-322
-
-
Stowens, D.W.1
Teitelbaum, S.L.2
Kahn, A.J.3
Barranger, J.A.4
-
19
-
-
60349121023
-
Lumbar spine peak bone mass and bone turnover in men and women: A longitudinal study
-
18629566 10.1007/s00198-008-0672-5 1:STN:280:DC%2BD1M7jtlSmug%3D%3D
-
Walsh JS, Henry YM, Fatayerji D, Eastell R (2009) Lumbar spine peak bone mass and bone turnover in men and women: a longitudinal study. Osteoporos Int 20:355-362
-
(2009)
Osteoporos Int
, vol.20
, pp. 355-362
-
-
Walsh, J.S.1
Henry, Y.M.2
Fatayerji, D.3
Eastell, R.4
-
20
-
-
0037159549
-
Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5years of treatment: A report from the Gaucher Registry
-
12133749 10.1016/S0002-9343(02)01150-6 1:CAS:528:DC%2BD38XlsVKru74%3D
-
Weinreb NJ, Charrow J, Andersson HC, Kaplan P, Kolodny EH, Mistry P, Pastores G, Rosenbloom BE, Scott CR, Wappner RS, Zimran A (2002) Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5years of treatment: a report from the Gaucher Registry. Am J Med 113:112-119
-
(2002)
Am J Med
, vol.113
, pp. 112-119
-
-
Weinreb, N.J.1
Charrow, J.2
Andersson, H.C.3
Kaplan, P.4
Kolodny, E.H.5
Mistry, P.6
Pastores, G.7
Rosenbloom, B.E.8
Scott, C.R.9
Wappner, R.S.10
Zimran, A.11
-
21
-
-
0026465017
-
Gaucher disease. Clinical, laboratory, radiologic, and genetic features of 53 patients
-
1435229 10.1097/00005792-199211000-00002 1:STN:280:DyaK3s%2FmtF2kug%3D%3D
-
Zimran A, Kay A, Gelbart T, Garver P, Thurston D, Saven A, Beutler E (1992) Gaucher disease. Clinical, laboratory, radiologic, and genetic features of 53 patients. Medicine 71:337-353
-
(1992)
Medicine
, vol.71
, pp. 337-353
-
-
Zimran, A.1
Kay, A.2
Gelbart, T.3
Garver, P.4
Thurston, D.5
Saven, A.6
Beutler, E.7
|