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Journal Abstract Search

326 related items for PubMed ID: 31912638

  • 1. 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
    [Abstract] [Full Text] [Related]

  • 2. Treating lysosomal storage disorders: What have we learnt?
    Lachmann RH.
    J Inherit Metab Dis; 2020 Jan; 43(1):125-132. PubMed ID: 31140601
    [Abstract] [Full Text] [Related]

  • 3. 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 Jan; 34(4 Suppl. 2):107-119. SPECIAL ISSUE: FOCUS ON PEDIATRIC CARDIOLOGY. PubMed ID: 33000609
    [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 Jan; 16(39):1-543. PubMed ID: 23089251
    [Abstract] [Full Text] [Related]

  • 5. Demographic characteristics and distribution of lysosomal storage disorder subtypes in Eastern China.
    Chen X, Qiu W, Ye J, Han L, Gu X, Zhang H.
    J Hum Genet; 2016 Apr; 61(4):345-9. PubMed ID: 26740238
    [Abstract] [Full Text] [Related]

  • 6. Real-world patient data on immunity and COVID-19 status of patients with MPS, Gaucher, and Pompe diseases from Turkey.
    Kilavuz S, Kor D, Bulut FD, Serbes M, Karagoz D, Altıntas DU, Bisgin A, Seydaoğlu G, Mungan HNO.
    Arch Pediatr; 2022 Aug; 29(6):415-423. PubMed ID: 35705384
    [Abstract] [Full Text] [Related]

  • 7. Treatable lysosomal storage diseases in the advent of disease-specific therapy.
    Peters H, Ellaway C, Nicholls K, Reardon K, Szer J.
    Intern Med J; 2020 Nov; 50 Suppl 4():5-27. PubMed ID: 33210402
    [Abstract] [Full Text] [Related]

  • 8. [Enzyme replacement therapy for lysosomal storage disorders].
    Valayannopoulos V, Brassier A, Chabli A, Caillaud C, Lemoine M, Odent T, Arnoux JB, de Lonlay P.
    Arch Pediatr; 2011 Oct; 18(10):1119-23. PubMed ID: 21873040
    [Abstract] [Full Text] [Related]

  • 9. Opinions of adults affected with later-onset lysosomal storage diseases regarding newborn screening: A qualitative study.
    Lisi EC, Ali N.
    J Genet Couns; 2021 Dec; 30(6):1544-1558. PubMed ID: 33938615
    [Abstract] [Full Text] [Related]

  • 10. Less Is More: Substrate Reduction Therapy for Lysosomal Storage Disorders.
    Coutinho MF, Santos JI, Alves S.
    Int J Mol Sci; 2016 Jul 04; 17(7):. PubMed ID: 27384562
    [Abstract] [Full Text] [Related]

  • 11. Enzyme replacement therapy for lysosomal storage diseases.
    Ohashi T.
    Pediatr Endocrinol Rev; 2012 Oct 04; 10 Suppl 1():26-34. PubMed ID: 23330243
    [Abstract] [Full Text] [Related]

  • 12.
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  • 13. 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 04; 8(1):9-25. PubMed ID: 24433521
    [Abstract] [Full Text] [Related]

  • 14. Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.
    Rohrbach M, Clarke JT.
    Drugs; 2007 Jan 04; 67(18):2697-716. PubMed ID: 18062719
    [Abstract] [Full Text] [Related]

  • 15. New prospects for the treatment of lysosomal storage diseases.
    Schiffmann R, Brady RO.
    Drugs; 2002 Jan 04; 62(5):733-42. PubMed ID: 11929328
    [Abstract] [Full Text] [Related]

  • 16. 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 21; 17(1):383. PubMed ID: 36271424
    [Abstract] [Full Text] [Related]

  • 17. [Treatment prospects of lysosomal storage disorders].
    Reismann P, Tulassay Z.
    Orv Hetil; 2008 Jun 22; 149(25):1171-9. PubMed ID: 18547894
    [Abstract] [Full Text] [Related]

  • 18. Neurological manifestations in lysosomal storage disorders - from pathology to first therapeutic possibilities.
    Hoffmann B, Mayatepek E.
    Neuropediatrics; 2005 Oct 22; 36(5):285-9. PubMed ID: 16217702
    [Abstract] [Full Text] [Related]

  • 19. Patients' perspectives on newborn screening for later-onset lysosomal storage diseases.
    Lisi EC, Gillespie S, Laney D, Ali N.
    Mol Genet Metab; 2016 Sep 22; 119(1-2):109-14. PubMed ID: 27591925
    [Abstract] [Full Text] [Related]

  • 20. Gene therapy for the lysosomal storage disorders.
    Cabrera-Salazar MA, Novelli E, Barranger JA.
    Curr Opin Mol Ther; 2002 Aug 22; 4(4):349-58. PubMed ID: 12222873
    [Abstract] [Full Text] [Related]

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