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 *

234 related articles for article (PubMed ID: 21162599)

  • 21. Pediatric Rheumatology Collaborative Study Group - over four decades of pivotal clinical drug research in pediatric rheumatology.
    Brunner HI; Rider LG; Kingsbury DJ; Co D; Schneider R; Goldmuntz E; Onel KB; Giannini EH; Lovell DJ;
    Pediatr Rheumatol Online J; 2018 Jul; 16(1):45. PubMed ID: 29996857
    [TBL] [Abstract][Full Text] [Related]  

  • 22. Enzyme replacement and beyond.
    Desnick RJ
    J Inherit Metab Dis; 2001 Apr; 24(2):251-65. PubMed ID: 11405344
    [TBL] [Abstract][Full Text] [Related]  

  • 23. 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]  

  • 24. Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction.
    Kishnani PS; Dickson PI; Muldowney L; Lee JJ; Rosenberg A; Abichandani R; Bluestone JA; Burton BK; Dewey M; Freitas A; Gavin D; Griebel D; Hogan M; Holland S; Tanpaiboon P; Turka LA; Utz JJ; Wang YM; Whitley CB; Kazi ZB; Pariser AR
    Mol Genet Metab; 2016 Feb; 117(2):66-83. PubMed ID: 26597321
    [TBL] [Abstract][Full Text] [Related]  

  • 25. Natural History, Trial Readiness and Gene Discovery: Advances in Patient Registries for Neuromuscular Disease.
    Thompson R; Robertson A; Lochmüller H
    Adv Exp Med Biol; 2017; 1031():97-124. PubMed ID: 29214567
    [TBL] [Abstract][Full Text] [Related]  

  • 26. A chaperone-mediated approach to enzyme enhancement as a therapeutic option for the lysosomal storage disorders.
    Pastores GM; Sathe S
    Drugs R D; 2006; 7(6):339-48. PubMed ID: 17073517
    [TBL] [Abstract][Full Text] [Related]  

  • 27. Impact of biobanks on research outcomes in rare diseases: a systematic review.
    Garcia M; Downs J; Russell A; Wang W
    Orphanet J Rare Dis; 2018 Nov; 13(1):202. PubMed ID: 30419920
    [TBL] [Abstract][Full Text] [Related]  

  • 28. Enzyme reconstitution/replacement therapy for lysosomal storage diseases.
    Burrow TA; Hopkin RJ; Leslie ND; Tinkle BT; Grabowski GA
    Curr Opin Pediatr; 2007 Dec; 19(6):628-35. PubMed ID: 18025928
    [TBL] [Abstract][Full Text] [Related]  

  • 29. Cardiac involvement in Lysosomal Storage Diseases.
    Sestito S; Parisi F; Tallarico V; Tarsitano F; Roppa K; Pensabene L; Chimenz R; Ceravolo G; Calabrò MP; De Sarro R; Moricca MT; Bonapace G; Concolino D
    J Biol Regul Homeost Agents; 2020; 34(4 Suppl. 2):107-119. SPECIAL ISSUE: FOCUS ON PEDIATRIC CARDIOLOGY. PubMed ID: 33000609
    [TBL] [Abstract][Full Text] [Related]  

  • 30. Enzyme Replacement Therapy: A Review and Its Role in Treating Lysosomal Storage Diseases.
    Li M
    Pediatr Ann; 2018 May; 47(5):e191-e197. PubMed ID: 29750286
    [TBL] [Abstract][Full Text] [Related]  

  • 31. The effectiveness of internet-based e-learning on clinician behavior and patient outcomes: a systematic review protocol.
    Sinclair P; Kable A; Levett-Jones T
    JBI Database System Rev Implement Rep; 2015 Jan; 13(1):52-64. PubMed ID: 26447007
    [TBL] [Abstract][Full Text] [Related]  

  • 32. Personalized Pharmacoperones for Lysosomal Storage Disorder: Approach for Next-Generation Treatment.
    Haneef SA; Doss CG
    Adv Protein Chem Struct Biol; 2016; 102():225-65. PubMed ID: 26827607
    [TBL] [Abstract][Full Text] [Related]  

  • 33. Proposal for the development of biologics in pediatric rheumatology.
    Mori M; Nakagawa M; Tsuchida N; Kawada K; Sato J; Sakiyama M; Hirano S; Sato K; Nakamura H
    Pediatr Int; 2018 Feb; 60(2):108-114. PubMed ID: 29288517
    [TBL] [Abstract][Full Text] [Related]  

  • 34. Behavioural modification interventions for medically unexplained symptoms in primary care: systematic reviews and economic evaluation.
    Leaviss J; Davis S; Ren S; Hamilton J; Scope A; Booth A; Sutton A; Parry G; Buszewicz M; Moss-Morris R; White P
    Health Technol Assess; 2020 Sep; 24(46):1-490. PubMed ID: 32975190
    [TBL] [Abstract][Full Text] [Related]  

  • 35. Gene therapy for lysosomal storage diseases: progress, challenges and future prospects.
    Seregin SS; Amalfitano A
    Curr Pharm Des; 2011; 17(24):2558-74. PubMed ID: 21774776
    [TBL] [Abstract][Full Text] [Related]  

  • 36. Pediatric cardiovascular safety: challenges in drug and device development and clinical application.
    Bates KE; Vetter VL; Li JS; Cummins S; Aguel F; Almond C; Dubin AM; Elia J; Finkle J; Hausner EA; Joseph F; Karkowsky AM; Killeen M; Lemacks J; Mathis L; McMahon AW; Pinnow E; Rodriguez I; Stockbridge NL; Stockwell M; Tassinari M; Krucoff MW
    Am Heart J; 2012 Oct; 164(4):481-92. PubMed ID: 23067905
    [TBL] [Abstract][Full Text] [Related]  

  • 37. Therapeutic approaches for lysosomal storage diseases: a patent update.
    Urbanelli L; Sagini K; Polidoro M; Brozzi A; Magini A; Emiliani C
    Recent Pat CNS Drug Discov; 2013 Aug; 8(2):91-109. PubMed ID: 23713988
    [TBL] [Abstract][Full Text] [Related]  

  • 38. Patient Registries: An Underused Resource for Medicines Evaluation : Operational proposals for increasing the use of patient registries in regulatory assessments.
    McGettigan P; Alonso Olmo C; Plueschke K; Castillon M; Nogueras Zondag D; Bahri P; Kurz X; Mol PGM
    Drug Saf; 2019 Nov; 42(11):1343-1351. PubMed ID: 31302896
    [TBL] [Abstract][Full Text] [Related]  

  • 39. Skeletal abnormalities in lysosomal storage diseases.
    Stevenson DA; Steiner RD
    Pediatr Endocrinol Rev; 2013 Jun; 10 Suppl 2():406-16. PubMed ID: 23858624
    [TBL] [Abstract][Full Text] [Related]  

  • 40. The DM-scope registry: a rare disease innovative framework bridging the gap between research and medical care.
    De Antonio M; Dogan C; Daidj F; Eymard B; Puymirat J; Mathieu J; Gagnon C; Katsahian S; ; Hamroun D; Bassez G
    Orphanet J Rare Dis; 2019 Jun; 14(1):122. PubMed ID: 31159885
    [TBL] [Abstract][Full Text] [Related]  

    [Previous]   [Next]    [New Search]
    of 12.