160 related articles for article (PubMed ID: 38183145)
1. Development and evaluation of a patient-reported outcome measure specific for Gaucher disease with or without neurological symptoms in Japan.
Narita A; Koto Y; Noto S; Okada M; Ono M; Baba T; Sagara R; Sakai N
Orphanet J Rare Dis; 2024 Jan; 19(1):11. PubMed ID: 38183145
[TBL] [Abstract][Full Text] [Related]
2. Burden of caregivers of patients with neuronopathic and non-neuronopathic Gaucher disease in Japan: A survey-based study.
Koto Y; Narita A; Noto S; Okada M; Ono M; Baba T; Sagara R; Sakai N
Mol Genet Metab Rep; 2023 Sep; 36():100994. PubMed ID: 37576570
[TBL] [Abstract][Full Text] [Related]
3. Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials.
Elstein D; Belmatoug N; Deegan P; Göker-Alpan Ö; Hughes DA; Schwartz IVD; Weinreb N; Bonner N; Panter C; Fountain D; Lenny A; Longworth L; Miller R; Shah K; Schenk J; Sen R; Zimran A
Orphanet J Rare Dis; 2022 Jan; 17(1):9. PubMed ID: 34991656
[TBL] [Abstract][Full Text] [Related]
4. Qualitative analysis of patient interviews on the burden of neuronopathic Gaucher disease in Japan.
Koto Y; Narita A; Noto S; Ono M; Hamada AL; Sakai N
Orphanet J Rare Dis; 2022 Jul; 17(1):280. PubMed ID: 35854314
[TBL] [Abstract][Full Text] [Related]
5. A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative.
Collin-Histed T; Stoodley M; Beusterien K; Elstein D; Jaffe DH; Revel-Vilk S; Davies EH;
Orphanet J Rare Dis; 2023 Jul; 18(1):195. PubMed ID: 37480076
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. Patient reported outcome measures in a large cohort of patients with type 1 Gaucher disease.
Dinur T; Istaiti M; Frydman D; Becker-Cohen M; Szer J; Zimran A; Revel-Vilk S
Orphanet J Rare Dis; 2020 Oct; 15(1):284. PubMed ID: 33050940
[TBL] [Abstract][Full Text] [Related]
8. Novel hyperkinetic dystonia-like manifestation and neurological disease course of Swedish Gaucher patients.
Machaczka M; Paucar M; Björkvall CK; Smith NJC; Cox TM; Forsgren L; Svenningsson P
Blood Cells Mol Dis; 2018 Feb; 68():86-92. PubMed ID: 27789132
[TBL] [Abstract][Full Text] [Related]
9. 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]
10. 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]
11. Qualitative exploration of the renal stone patients' experience and development of the renal stone-specific patient-reported outcome measure.
Ragab M; Baldin N; Collie J; Tran MGB; Al-Hayek S; S Parsy K; Armitage J; Wiseman O
BJU Int; 2020 Jan; 125(1):123-132. PubMed ID: 31313440
[TBL] [Abstract][Full Text] [Related]
12. Gaucher disease and its treatment options.
Bennett LL; Mohan D
Ann Pharmacother; 2013 Sep; 47(9):1182-93. PubMed ID: 24259734
[TBL] [Abstract][Full Text] [Related]
13. Patient reported outcomes of patients with Gaucher disease type 1 treated with eliglustat in real-world settings: The ELIPRO study.
Camou F; Lagadec A; Coutinho A; Berger MG; Cador-Rousseau B; Gaches F; Belmatoug N
Mol Genet Metab; 2023 Nov; 140(3):107667. PubMed ID: 37597334
[TBL] [Abstract][Full Text] [Related]
14. A characterization of Gaucher iPS-derived astrocytes: Potential implications for Parkinson's disease.
Aflaki E; Stubblefield BK; McGlinchey RP; McMahon B; Ory DS; Sidransky E
Neurobiol Dis; 2020 Feb; 134():104647. PubMed ID: 31669751
[TBL] [Abstract][Full Text] [Related]
15. Urinary Stones and Intervention Quality of Life (USIQoL): Development and Validation of a New Core Universal Patient-reported Outcome Measure for Urinary Calculi.
Joshi HB; Johnson H; Pietropaolo A; Raja A; Joyce AD; Somani B; Philip J; Biyani CS; Pickles T
Eur Urol Focus; 2022 Jan; 8(1):283-290. PubMed ID: 33423970
[TBL] [Abstract][Full Text] [Related]
16. The D409H variant in GBA1: Challenges in predicting the Gaucher phenotype in the newborn screening era.
Gleason AM; D'Souza A; Ryan E; Grochowsky AR; Carter CR; Goker-Alpan O; Lopez G; Tayebi N; Sidransky E
Am J Med Genet A; 2023 Jul; 191(7):1783-1791. PubMed ID: 37042183
[TBL] [Abstract][Full Text] [Related]
17. Development and validation of a set of patient reported outcome measures to assess effectiveness of asthma prophylaxis.
Guruparan Y; Navaratinaraja TS; Selvaratnam G; Gunawardena N; Sri Ranganathan S
BMC Pulm Med; 2021 Sep; 21(1):295. PubMed ID: 34535111
[TBL] [Abstract][Full Text] [Related]
18. Comprehensive and long-term outcomes of enzyme replacement therapy followed by stem cell transplantation in children with Gaucher disease type 1 and 3.
Anurathapan U; Tim-Aroon T; Zhang W; Sanpote W; Wongrungsri S; Khunin N; Chutipongtanate S; Chirdkiatgumchai V; Ngiwsara L; Jaovisidha S; Khongkraparn A; Pakakasama S; Svasti J; Setchell KDR; Wattanasirichaigoon D; Hongeng S
Pediatr Blood Cancer; 2023 Mar; 70(3):e30149. PubMed ID: 36562549
[TBL] [Abstract][Full Text] [Related]
19. Development and validation of the genitourinary syndrome of menopause symptoms and vaginal treatments acceptability questionnaire (GSM-SVTAQ): An electronic patient-reported outcomes measure.
Hassanein MM; Huri HZ; Baig K; Abduelkarem AR; Al-Momani M
Int J Gynaecol Obstet; 2024 Feb; 164(2):613-623. PubMed ID: 37702968
[TBL] [Abstract][Full Text] [Related]
20. Patient reported outcome measure-haemorrhoidal impact and satisfaction score (PROM-HISS): Development, reliability and construct validity.
Kuiper SZ; Kimman ML; Van Tol RR; Waardenburg SF; Van Kuijk SMJ; Dirksen CD; Breukink SO
Colorectal Dis; 2022 Aug; 24(8):992-999. PubMed ID: 35119715
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
