177 related articles for article (PubMed ID: 35782609)
61. Long term effects of enzyme replacement therapy in an Italian cohort of type 3 Gaucher patients.
Sechi A; Deroma L; Dardis A; Ciana G; Bertin N; Concolino D; Linari S; Perria C; Bembi B
Mol Genet Metab; 2014 Nov; 113(3):213-8. PubMed ID: 25127542
[TBL] [Abstract][Full Text] [Related]
62. A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1.
Lukina E; Watman N; Arreguin EA; Banikazemi M; Dragosky M; Iastrebner M; Rosenbaum H; Phillips M; Pastores GM; Rosenthal DI; Kaper M; Singh T; Puga AC; Bonate PL; Peterschmitt MJ
Blood; 2010 Aug; 116(6):893-9. PubMed ID: 20439622
[TBL] [Abstract][Full Text] [Related]
63. Hematopoietic stem cell transplantation or enzyme replacement therapy in Gaucher disease type 3.
Høj A; Ørngreen MC; Naume MM; Lund AM
Mol Genet Metab; 2024 Jun; 142(4):108515. PubMed ID: 38909587
[TBL] [Abstract][Full Text] [Related]
64. Pulmonary Involvement Responsive to Enzyme Replacement Therapy in an Elderly Patient with Gaucher Disease.
Vellas D; Gramont B; Grange R; Cathébras P
Eur J Case Rep Intern Med; 2021; 8(9):002802. PubMed ID: 34671576
[TBL] [Abstract][Full Text] [Related]
65. Impact of Long-Term Enzyme Replacement Therapy on Glucosylsphingosine (Lyso-Gb1) Values in Patients with Type 1 Gaucher Disease: Statistical Models for Comparing Three Enzymatic Formulations.
Dinur T; Grittner U; Revel-Vilk S; Becker-Cohen M; Istaiti M; Cozma C; Rolfs A; Zimran A
Int J Mol Sci; 2021 Jul; 22(14):. PubMed ID: 34299318
[TBL] [Abstract][Full Text] [Related]
66. Glucosylsphingosine (lyso-Gb1) as a Biomarker for Monitoring Treated and Untreated Children with Gaucher Disease.
Hurvitz N; Dinur T; Becker-Cohen M; Cozma C; Hovakimyan M; Oppermann S; Demuth L; Rolfs A; Abramov A; Zimran A; Revel-Vilk S
Int J Mol Sci; 2019 Jun; 20(12):. PubMed ID: 31234327
[TBL] [Abstract][Full Text] [Related]
67. Clinical response to eliglustat in treatment-naïve patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase-treated patients enrolled in the International Collaborative Gaucher Group Gaucher Registry.
Ibrahim J; Underhill LH; Taylor JS; Angell J; Peterschmitt MJ
Mol Genet Metab Rep; 2016 Sep; 8():17-9. PubMed ID: 27408819
[TBL] [Abstract][Full Text] [Related]
68. Pregnancy outcome in women with Gaucher disease type 1 who had unplanned pregnancies during eliglustat clinical trials.
Lukina E; Balwani M; Belmatoug N; Watman N; Hughes D; Gaemers SJM; Foster MC; Lewis G; Peterschmitt MJ
JIMD Rep; 2021 Jan; 57(1):76-84. PubMed ID: 33473343
[TBL] [Abstract][Full Text] [Related]
69. Enzyme replacement therapy and bony changes in Egyptian paediatric Gaucher disease patients.
El-Beshlawy A; Ragab L; Youssry I; Yakout K; El-Kiki H; Eid K; Mansour IM; Abd El-Hamid S; Yang M; Mistry PK
J Inherit Metab Dis; 2006 Feb; 29(1):92-8. PubMed ID: 16601874
[TBL] [Abstract][Full Text] [Related]
70. Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment.
Lukina E; Watman N; Dragosky M; Pastores GM; Arreguin EA; Rosenbaum H; Zimran A; Angell J; Ross L; Puga AC; Peterschmitt JM
Blood Cells Mol Dis; 2014 Dec; 53(4):274-6. PubMed ID: 24835462
[TBL] [Abstract][Full Text] [Related]
71. Effect of Substrate Reduction Therapy in Comparison to Enzyme Replacement Therapy on Immune Aspects and Bone Involvement in Gaucher Disease.
Limgala RP; Goker-Alpan O
Biomolecules; 2020 Mar; 10(4):. PubMed ID: 32244296
[TBL] [Abstract][Full Text] [Related]
72. Nine-year experience in Gaucher disease diagnosis at the Spanish reference center Fundación Jiménez Díaz.
Ortiz-Cabrera NV; Gallego-Merlo J; Vélez-Monsalve C; de Nicolas R; Mas SF; Ayuso C; Trujillo-Tiebas MJ
Mol Genet Metab Rep; 2016 Dec; 9():79-85. PubMed ID: 27872820
[TBL] [Abstract][Full Text] [Related]
73. Chitotriosidase variants in patients with Gaucher disease. Implications for diagnosis and therapeutic monitoring.
Irún P; Alfonso P; Aznarez S; Giraldo P; Pocovi M
Clin Biochem; 2013 Dec; 46(18):1804-7. PubMed ID: 24060732
[TBL] [Abstract][Full Text] [Related]
74. Gaucher disease: clinical phenotypes and refining GBA mutational spectrum in Thai patients.
Phetthong T; Tim-Aroon T; Khongkraparn A; Noojarern S; Kuptanon C; Wichajarn K; Sathienkijkanchai A; Suphapeetiporn K; Charoenkwan P; Tantiworawit A; Noentong N; Wattanasirichaigoon D
Orphanet J Rare Dis; 2021 Dec; 16(1):519. PubMed ID: 34930372
[TBL] [Abstract][Full Text] [Related]
75. Treatment-naive and post-treatment glucosylsphingosine (lyso-GL1) levels in a cohort of pediatric patients with Gaucher disease.
Rasmussen CA; Quadri A; Vucko E; Kim K; Hickey R; Baker JJ; Charrow J; Prada CE
Mol Genet Metab; 2024 Jan; 141(1):107736. PubMed ID: 38000346
[TBL] [Abstract][Full Text] [Related]
76. Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy.
Marshall J; McEachern KA; Chuang WL; Hutto E; Siegel CS; Shayman JA; Grabowski GA; Scheule RK; Copeland DP; Cheng SH
J Inherit Metab Dis; 2010 Jun; 33(3):281-9. PubMed ID: 20336375
[TBL] [Abstract][Full Text] [Related]
77. Effect of enzyme replacement therapy with imiglucerase on BMD in type 1 Gaucher disease.
Wenstrup RJ; Kacena KA; Kaplan P; Pastores GM; Prakash-Cheng A; Zimran A; Hangartner TN
J Bone Miner Res; 2007 Jan; 22(1):119-26. PubMed ID: 17032149
[TBL] [Abstract][Full Text] [Related]
78. Clinical-genetic characteristics and treatment outcomes of Turkish children with Gaucher disease type 1 and type 3: A sixteen year single-center experience.
Gumus E; Karhan AN; Hizarcioglu-Gulsen H; Demir H; Ozen H; Saltik Temizel IN; Dokmeci Emre S; Yuce A
Eur J Med Genet; 2021 Nov; 64(11):104339. PubMed ID: 34500086
[TBL] [Abstract][Full Text] [Related]
79. Promising Effect of High Dose Ambroxol Treatment on Neurocognition and Motor Development in a Patient With Neuropathic Gaucher Disease 2.
Aries C; Lohmöller B; Tiede S; Täuber K; Hartmann G; Rudolph C; Muschol N
Front Neurol; 2022; 13():907317. PubMed ID: 35734474
[TBL] [Abstract][Full Text] [Related]
80. Different dose-dependent correction of MIP-1beta and chitotriosidase during initial enzyme replacement therapy.
van Breemen MJ; de Fost M; Maas M; Wiersma MG; Hollak CE; Poll LW; Vom Dahl S; Boot RG; Aerts JM
J Inherit Metab Dis; 2009 Apr; 32(2):274-9. PubMed ID: 19255873
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]