132 related articles for article (PubMed ID: 38930117)
1. Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry.
Elstein D; Belmatoug N; Bembi B; Deegan P; Fernandez-Sasso D; Giraldo P; Göker-Alpan Ö; Hughes D; Lau H; Lukina E; Revel-Vilk S; Schwartz IVD; Istaiti M; Botha J; Gadir N; Schenk J; Zimran A;
J Clin Med; 2024 Jun; 13(12):. PubMed ID: 38930117
[No Abstract] [Full Text] [Related]
2. Velaglucerase alfa for the management of type 1 Gaucher disease.
Morris JL
Clin Ther; 2012 Feb; 34(2):259-71. PubMed ID: 22264444
[TBL] [Abstract][Full Text] [Related]
3. Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry.
Deegan P; Lau H; Elstein D; Fernandez-Sasso D; Giraldo P; Hughes D; Zimran A; Istaiti M; Gadir N; Botha J; Revel-Vilk S;
J Clin Med; 2024 May; 13(10):. PubMed ID: 38792324
[No Abstract] [Full Text] [Related]
4. Characteristics of 26 patients with type 3 Gaucher disease: A descriptive analysis from the Gaucher Outcome Survey.
Schwartz IVD; Göker-Alpan Ö; Kishnani PS; Zimran A; Renault L; Panahloo Z; Deegan P;
Mol Genet Metab Rep; 2018 Mar; 14():73-79. PubMed ID: 29326879
[TBL] [Abstract][Full Text] [Related]
5. Enzyme replacement and substrate reduction therapy for Gaucher disease.
Shemesh E; Deroma L; Bembi B; Deegan P; Hollak C; Weinreb NJ; Cox TM
Cochrane Database Syst Rev; 2015 Mar; 2015(3):CD010324. PubMed ID: 25812601
[TBL] [Abstract][Full Text] [Related]
6. Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS).
Zimran A; Belmatoug N; Bembi B; Deegan P; Elstein D; Fernandez-Sasso D; Giraldo P; Goker-Alpan O; Lau H; Lukina E; Panahloo Z; Schwartz IVD;
Am J Hematol; 2018 Feb; 93(2):205-212. PubMed ID: 29090476
[TBL] [Abstract][Full Text] [Related]
7. Treatment patterns from 647 patients with Gaucher disease: An analysis from the Gaucher Outcome Survey.
Deegan P; Fernandez-Sasso D; Giraldo P; Lau H; Panahloo Z; Zimran A
Blood Cells Mol Dis; 2018 Feb; 68():218-225. PubMed ID: 27829541
[TBL] [Abstract][Full Text] [Related]
8. A multicenter, open-label extension study of velaglucerase alfa in Japanese patients with Gaucher disease: Results after a cumulative treatment period of 24months.
Ida H; Tanaka A; Matsubayashi T; Murayama K; Hongo T; Lee HM; Mellgard B
Blood Cells Mol Dis; 2016 Jul; 59():140-7. PubMed ID: 27241455
[TBL] [Abstract][Full Text] [Related]
9. Long-term safety and effectiveness of velaglucerase alfa in Gaucher disease: 6-year interim analysis of a post-marketing surveillance in Japan.
Sagara R; Ishigaki M; Otsuka M; Murayama K; Ida H; Fernandez J
Orphanet J Rare Dis; 2021 Dec; 16(1):502. PubMed ID: 34863216
[TBL] [Abstract][Full Text] [Related]
10. Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS).
Titievsky L; Schuster T; Wang R; Younus M; Palladino A; Quazi K; Wajnrajch MP; Hernandez B; Becker PS; Weinreb NJ; Chambers C; Mansfield R; Taylor L; Tseng LJ; Kaplan P
Orphanet J Rare Dis; 2022 Apr; 17(1):145. PubMed ID: 35365177
[TBL] [Abstract][Full Text] [Related]
11. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS).
Hughes DA; Deegan P; Giraldo P; Göker-Alpan Ö; Lau H; Lukina E; Revel-Vilk S; Scarpa M; Botha J; Gadir N; Zimran A;
J Clin Med; 2022 Aug; 11(17):. PubMed ID: 36079085
[TBL] [Abstract][Full Text] [Related]
12. Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase.
Smith L; Rhead W; Charrow J; Shankar SP; Bavdekar A; Longo N; Mardach R; Harmatz P; Hangartner T; Lee HM; Crombez E; Pastores GM
Mol Genet Metab; 2016 Feb; 117(2):164-71. PubMed ID: 26043810
[TBL] [Abstract][Full Text] [Related]
13. Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment.
Weinreb NJ; Camelo JS; Charrow J; McClain MR; Mistry P; Belmatoug N;
Mol Genet Metab; 2021 Feb; 132(2):100-111. PubMed ID: 33485799
[TBL] [Abstract][Full Text] [Related]
14. Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease.
Elstein D; Mehta A; Hughes DA; Giraldo P; Charrow J; Smith L; Shankar SP; Hangartner TN; Kunes Y; Wang N; Crombez E; Zimran A
Am J Hematol; 2015 Jul; 90(7):592-7. PubMed ID: 25776130
[TBL] [Abstract][Full Text] [Related]
15. Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry.
Beck M; Ramaswami U; Hernberg-Ståhl E; Hughes DA; Kampmann C; Mehta AB; Nicholls K; Niu DM; Pintos-Morell G; Reisin R; West ML; Schenk J; Anagnostopoulou C; Botha J; Giugliani R
Orphanet J Rare Dis; 2022 Jun; 17(1):238. PubMed ID: 35725623
[TBL] [Abstract][Full Text] [Related]
16. Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry.
El-Beshlawy A; Tylki-Szymanska A; Vellodi A; Belmatoug N; Grabowski GA; Kolodny EH; Batista JL; Cox GF; Mistry PK
Mol Genet Metab; 2017; 120(1-2):47-56. PubMed ID: 28040394
[TBL] [Abstract][Full Text] [Related]
17. The budget impact of enzyme replacement therapy in type 1 Gaucher disease in the United States.
Farahbakhshian S; Inocencio TJ; Poorman G; Wright E; Pathak RR; Bullano M
J Med Econ; 2022; 25(1):755-761. PubMed ID: 35611840
[TBL] [Abstract][Full Text] [Related]
18. Reported outcomes of 453 pregnancies in patients with Gaucher disease: An analysis from the Gaucher outcome survey.
Lau H; Belmatoug N; Deegan P; Goker-Alpan O; Schwartz IVD; Shankar SP; Panahloo Z; Zimran A
Blood Cells Mol Dis; 2018 Feb; 68():226-231. PubMed ID: 27839985
[TBL] [Abstract][Full Text] [Related]
19. Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trials.
Hughes DA; Gonzalez DE; Lukina EA; Mehta A; Kabra M; Elstein D; Kisinovsky I; Giraldo P; Bavdekar A; Hangartner TN; Wang N; Crombez E; Zimran A
Am J Hematol; 2015 Jul; 90(7):584-91. PubMed ID: 25801797
[TBL] [Abstract][Full Text] [Related]
20. Baseline characteristics of 32 patients with Gaucher disease who were treated with imiglucerase: South African data from the International Collaborative Gaucher Group (ICGG) Gaucher Registry.
Sevittz H; Laher F; Varughese ST; Nel M; McMaster A; Jacobson BF
S Afr Med J; 2022 Feb; 112(1):13518. PubMed ID: 35140000
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]