These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

166 related articles for article (PubMed ID: 28029334)

  • 1. PATIENT-REPORTED OUTCOMES IN RARE LYSOSOMAL STORAGE DISEASES: KEY INFORMANT INTERVIEWS AND A SYSTEMATIC REVIEW PROTOCOL.
    Miller PA; Mulla SM; Adams-Webber T; Sivji Y; Guyatt GH; Johnston BC
    Int J Technol Assess Health Care; 2016 Jan; 32(6):393-399. PubMed ID: 28029334
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Limited responsiveness related to the minimal important difference of patient-reported outcomes in rare diseases.
    Johnston BC; Miller PA; Agarwal A; Mulla S; Khokhar R; De Oliveira K; Hitchcock CL; Sadeghirad B; Mohiuddin M; Sekercioglu N; Seweryn M; Koperny M; Bala MM; Adams-Webber T; Granados A; Hamed A; Crawford MW; van der Ploeg AT; Guyatt GH
    J Clin Epidemiol; 2016 Nov; 79():10-21. PubMed ID: 27381737
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Patients' view on gene therapy development for lysosomal storage disorders: a qualitative study.
    Eskes ECB; Beishuizen CRL; Corazolla EM; van Middelaar T; Brands MMMG; Dekker H; van de Mheen E; Langeveld M; Hollak CEM; Sjouke B
    Orphanet J Rare Dis; 2022 Oct; 17(1):383. PubMed ID: 36271424
    [TBL] [Abstract][Full Text] [Related]  

  • 4. The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders.
    Wyatt K; Henley W; Anderson L; Anderson R; Nikolaou V; Stein K; Klinger L; Hughes D; Waldek S; Lachmann R; Mehta A; Vellodi A; Logan S
    Health Technol Assess; 2012; 16(39):1-543. PubMed ID: 23089251
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Pulmonary involvement in selected lysosomal storage diseases and the impact of enzyme replacement therapy: A state-of-the art review.
    Jezela-Stanek A; Chorostowska-Wynimko J; Tylki-Szymańska A
    Clin Respir J; 2020 May; 14(5):422-429. PubMed ID: 31912638
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Prevalence of patients with lysosomal storage disorders and peroxisomal disorders: A nationwide survey in Japan.
    Koto Y; Sakai N; Lee Y; Kakee N; Matsuda J; Tsuboi K; Shimozawa N; Okuyama T; Nakamura K; Narita A; Kobayashi H; Uehara R; Nakamura Y; Kato K; Eto Y
    Mol Genet Metab; 2021 Jul; 133(3):277-288. PubMed ID: 34090759
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Folic acid supplementation and malaria susceptibility and severity among people taking antifolate antimalarial drugs in endemic areas.
    Crider K; Williams J; Qi YP; Gutman J; Yeung L; Mai C; Finkelstain J; Mehta S; Pons-Duran C; Menéndez C; Moraleda C; Rogers L; Daniels K; Green P
    Cochrane Database Syst Rev; 2022 Feb; 2(2022):. PubMed ID: 36321557
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Neurological manifestations in lysosomal storage disorders - from pathology to first therapeutic possibilities.
    Hoffmann B; Mayatepek E
    Neuropediatrics; 2005 Oct; 36(5):285-9. PubMed ID: 16217702
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Newborn screening of lysosomal storage disorders.
    Marsden D; Levy H
    Clin Chem; 2010 Jul; 56(7):1071-9. PubMed ID: 20489136
    [TBL] [Abstract][Full Text] [Related]  

  • 10. A Comparative Study on the Alterations of Endocytic Pathways in Multiple Lysosomal Storage Disorders.
    Rappaport J; Manthe RL; Solomon M; Garnacho C; Muro S
    Mol Pharm; 2016 Feb; 13(2):357-368. PubMed ID: 26702793
    [TBL] [Abstract][Full Text] [Related]  

  • 11. The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment.
    Whittal A; Meregaglia M; Nicod E
    Patient; 2021 Sep; 14(5):485-503. PubMed ID: 33462774
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Therapeutic Role of Pharmacological Chaperones in Lysosomal Storage Disorders: A Review of the Evidence and Informed Approach to Reclassification.
    Keyzor I; Shohet S; Castelli J; Sitaraman S; Veleva-Rotse B; Weimer JM; Fox B; Willer T; Tuske S; Crathorne L; Belzar KJ
    Biomolecules; 2023 Aug; 13(8):. PubMed ID: 37627292
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Comprehensive review of statistical methods for analysing patient-reported outcomes (PROs) used as primary outcomes in randomised controlled trials (RCTs) published by the UK's
    Qian Y; Walters SJ; Jacques R; Flight L
    BMJ Open; 2021 Sep; 11(9):e051673. PubMed ID: 34489292
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Rare lysosomal disease registries: lessons learned over three decades of real-world evidence.
    Mistry PK; Kishnani P; Wanner C; Dong D; Bender J; Batista JL; Foster J
    Orphanet J Rare Dis; 2022 Oct; 17(1):362. PubMed ID: 36244992
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Treatment of lysosomal storage diseases: recent patents and future strategies.
    Ortolano S; Viéitez I; Navarro C; Spuch C
    Recent Pat Endocr Metab Immune Drug Discov; 2014 Jan; 8(1):9-25. PubMed ID: 24433521
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Expanding newborn screening for lysosomal disorders: opportunities and challenges.
    Waggoner DJ; Tan CA
    Dev Disabil Res Rev; 2011; 17(1):9-14. PubMed ID: 22447749
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Evaluation of patient involvement strategies in health technology assessment in Spain: the viewpoint of HTA researchers.
    Toledo-Chávarri A; Triñanes Pego Y; Rodrigo ER; Roteta NI; Novella-Arribas B; Vicente Edo MJ; Gavín Benavent P; Puigdomenech E; Molina Linde JM
    Int J Technol Assess Health Care; 2020 Sep; 37():e25. PubMed ID: 32914735
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Improving Interpretation of Evidence Relating to Quality of Life in Health Technology Assessments of Rare Disease Treatments.
    Nicod E; Lloyd AJ; Morel T; Meregaglia M; Upadhyaya S; Whittal A; Facey K; Drummond M
    Patient; 2023 Jan; 16(1):7-17. PubMed ID: 36217098
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Newborn screening for six lysosomal storage disorders in a cohort of Mexican patients: Three-year findings from a screening program in a closed Mexican health system.
    Navarrete-Martínez JI; Limón-Rojas AE; Gaytán-García MJ; Reyna-Figueroa J; Wakida-Kusunoki G; Delgado-Calvillo MDR; Cantú-Reyna C; Cruz-Camino H; Cervantes-Barragán DE
    Mol Genet Metab; 2017 May; 121(1):16-21. PubMed ID: 28302345
    [TBL] [Abstract][Full Text] [Related]  

  • 20.
    ; ; . PubMed ID:
    [No Abstract]   [Full Text] [Related]  

    [Next]    [New Search]
    of 9.