Results
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81.
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Prevalence of type 1 Gaucher disease in the United States. [electronic resource] by
- Weinreb, Neal J
- Andersson, Hans C
- Banikazemi, Maryam
- Barranger, John
- Beutler, Ernest
- Charrow, Joel
- Grabowski, Gregory A
- Hollak, Carla E M
- Kaplan, Paige
- Mankin, Henry
- Mistry, Pramod K
- Rosenbloom, Barry E
- Vom Dahl, Stephan
- Zimran, Ari
Producer: 20080306
In:
Archives of internal medicine vol. 168
Availability: No items available.
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82.
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Glucocerebrosidase 2 gene deletion rescues type 1 Gaucher disease. [electronic resource] by
- Mistry, Pramod K
- Liu, Jun
- Sun, Li
- Chuang, Wei-Lien
- Yuen, Tony
- Yang, Ruhua
- Lu, Ping
- Zhang, Kate
- Li, Jianhua
- Keutzer, Joan
- Stachnik, Agnes
- Mennone, Albert
- Boyer, James L
- Jain, Dhanpat
- Brady, Roscoe O
- New, Maria I
- Zaidi, Mone
Producer: 20140602
In:
Proceedings of the National Academy of Sciences of the United States of America vol. 111
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83.
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Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial. [electronic resource] by
- Mistry, Pramod K
- Lukina, Elena
- Ben Turkia, Hadhami
- Shankar, Suma P
- Baris, Hagit
- Ghosn, Marwan
- Mehta, Atul
- Packman, Seymour
- Pastores, Gregory
- Petakov, Milan
- Assouline, Sarit
- Balwani, Manisha
- Danda, Sumita
- Hadjiev, Evgueniy
- Ortega, Andres
- Gaemers, Sebastiaan J M
- Tayag, Regina
- Peterschmitt, M Judith
Producer: 20171024
In:
American journal of hematology vol. 92
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84.
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The Role of ARF6 in Biliary Atresia. [electronic resource] by
- Ningappa, Mylarappa
- So, Juhoon
- Glessner, Joseph
- Ashokkumar, Chethan
- Ranganathan, Sarangarajan
- Min, Jun
- Higgs, Brandon W
- Sun, Qing
- Haberman, Kimberly
- Schmitt, Lori
- Vilarinho, Silvia
- Mistry, Pramod K
- Vockley, Gerard
- Dhawan, Anil
- Gittes, George K
- Hakonarson, Hakon
- Jaffe, Ronald
- Subramaniam, Shankar
- Shin, Donghun
- Sindhi, Rakesh
Producer: 20160520
In:
PloS one vol. 10
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85.
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Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response. [electronic resource] by
- Dekker, Nick
- van Dussen, Laura
- Hollak, Carla E M
- Overkleeft, Herman
- Scheij, Saskia
- Ghauharali, Karen
- van Breemen, Mariëlle J
- Ferraz, Maria J
- Groener, Johanna E M
- Maas, Mario
- Wijburg, Frits A
- Speijer, Dave
- Tylki-Szymanska, Anna
- Mistry, Pramod K
- Boot, Rolf G
- Aerts, Johannes M
Producer: 20111220
In:
Blood vol. 118
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86.
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Hepatocellular carcinoma in Gaucher disease: an international case series. [electronic resource] by
- Regenboog, Martine
- van Dussen, Laura
- Verheij, Joanne
- Weinreb, Neal J
- Santosa, David
- Vom Dahl, Stephan
- Häussinger, Dieter
- Müller, Meike N
- Canbay, Ali
- Rigoldi, Miriam
- Piperno, Alberto
- Dinur, Tama
- Zimran, Ari
- Mistry, Pramod K
- Salah, Karima Yousfi
- Belmatoug, Nadia
- Kuter, David J
- Hollak, Carla E M
Producer: 20191107
In:
Journal of inherited metabolic disease vol. 41
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87.
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Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial. [electronic resource] by
- Mistry, Pramod K
- Lukina, Elena
- Ben Turkia, Hadhami
- Amato, Dominick
- Baris, Hagit
- Dasouki, Majed
- Ghosn, Marwan
- Mehta, Atul
- Packman, Seymour
- Pastores, Gregory
- Petakov, Milan
- Assouline, Sarit
- Balwani, Manisha
- Danda, Sumita
- Hadjiev, Evgueniy
- Ortega, Andres
- Shankar, Suma
- Solano, Maria Helena
- Ross, Leorah
- Angell, Jennifer
- Peterschmitt, M Judith
Producer: 20150303
In:
JAMA vol. 313
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88.
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Mutations in GBA2 cause autosomal-recessive cerebellar ataxia with spasticity. [electronic resource] by
- Hammer, Monia B
- Eleuch-Fayache, Ghada
- Schottlaender, Lucia V
- Nehdi, Houda
- Gibbs, J Raphael
- Arepalli, Sampath K
- Chong, Sean B
- Hernandez, Dena G
- Sailer, Anna
- Liu, Guoxiang
- Mistry, Pramod K
- Cai, Huaibin
- Shrader, Ginamarie
- Sassi, Celeste
- Bouhlal, Yosr
- Houlden, Henry
- Hentati, Fayçal
- Amouri, Rim
- Singleton, Andrew B
Producer: 20130404
In:
American journal of human genetics vol. 92
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89.
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Antigen-mediated regulation in monoclonal gammopathies and myeloma. [electronic resource] by
- Nair, Shiny
- Sng, Joel
- Boddupalli, Chandra Sekhar
- Seckinger, Anja
- Chesi, Marta
- Fulciniti, Mariateresa
- Zhang, Lin
- Rauniyar, Navin
- Lopez, Michael
- Neparidze, Natalia
- Parker, Terri
- Munshi, Nikhil C
- Sexton, Rachael
- Barlogie, Bart
- Orlowski, Robert
- Bergsagel, Leif
- Hose, Dirk
- Flavell, Richard A
- Mistry, Pramod K
- Meffre, Eric
- Dhodapkar, Madhav V
Producer: 20191120
In:
JCI insight vol. 3
Availability: No items available.
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90.
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Glucocerebrosidase gene-deficient mouse recapitulates Gaucher disease displaying cellular and molecular dysregulation beyond the macrophage. [electronic resource] by
- Mistry, Pramod K
- Liu, Jun
- Yang, Mei
- Nottoli, Timothy
- McGrath, James
- Jain, Dhanpat
- Zhang, Kate
- Keutzer, Joan
- Chuang, Wei-Lien
- Chuang, Wei-Lein
- Mehal, Wajahat Z
- Zhao, Hongyu
- Lin, Aiping
- Mane, Shrikant
- Liu, Xuan
- Peng, Yuan Z
- Li, Jian H
- Agrawal, Manasi
- Zhu, Ling-Ling
- Blair, Harry C
- Robinson, Lisa J
- Iqbal, Jameel
- Sun, Li
- Zaidi, Mone
Producer: 20101230
In:
Proceedings of the National Academy of Sciences of the United States of America vol. 107
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91.
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Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1. [electronic resource] by
- Mistry, Pramod K
- Balwani, Manisha
- Baris, Hagit N
- Turkia, Hadhami Ben
- Burrow, T Andrew
- Charrow, Joel
- Cox, Gerald F
- Danda, Sumita
- Dragosky, Marta
- Drelichman, Guillermo
- El-Beshlawy, Amal
- Fraga, Cristina
- Freisens, Selena
- Gaemers, Sebastiaan
- Hadjiev, Evgueniy
- Kishnani, Priya S
- Lukina, Elena
- Maison-Blanche, Pierre
- Martins, Ana Maria
- Pastores, Gregory
- Petakov, Milan
- Peterschmitt, M Judith
- Rosenbaum, Hanna
- Rosenbloom, Barry
- Underhill, Lisa H
- Cox, Timothy M
Producer: 20190218
In:
Blood cells, molecules & diseases vol. 71
Availability: No items available.
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92.
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Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients. [electronic resource] by
- Weinreb, Neal J
- Aggio, Mario C
- Andersson, Hans C
- Andria, Generoso
- Charrow, Joel
- Clarke, Joe T R
- Erikson, Anders
- Giraldo, Pilar
- Goldblatt, Jack
- Hollak, Carla
- Ida, Hiroyuki
- Kaplan, Paige
- Kolodny, Edwin H
- Mistry, Pramod
- Pastores, Gregory M
- Pires, Ricardo
- Prakash-Cheng, Ainu
- Prakesh-Cheng, Ainu
- Rosenbloom, Barry E
- Scott, C Ronald
- Sobreira, Elisa
- Tylki-Szymańska, Anna
- Vellodi, Ashok
- vom Dahl, Stephan
- Wappner, Rebecca S
- Zimran, Ari
Producer: 20050331
In:
Seminars in hematology vol. 41
Availability: No items available.
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93.
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Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1. [electronic resource] by
- Mistry, Pramod K
- Balwani, Manisha
- Baris, Hagit N
- Turkia, Hadhami Ben
- Burrow, T Andrew
- Charrow, Joel
- Cox, Gerald F
- Danda, Sumita
- Dragosky, Marta
- Drelichman, Guillermo
- El-Beshlawy, Amal
- Fraga, Cristina
- Freisens, Selena
- Gaemers, Sebastiaan
- Hadjiev, Evgueniy
- Kishnani, Priya S
- Lukina, Elena
- Maison-Blanche, Pierre
- Martins, Ana Maria
- Pastores, Gregory
- Petakov, Milan
- Peterschmitt, M Judith
- Rosenbaum, Hanna
- Rosenbloom, Barry
- Underhill, Lisa H
- Cox, Timothy M
Producer: 20200114
In:
Blood cells, molecules & diseases vol. 77
Availability: No items available.
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