BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

172 related articles for article (PubMed ID: 31481328)

  • 1. The End of the Beginning: The Journey to Molecular Therapies for Spinal Muscular Atrophy.
    Pacak CA; Kang PB
    Pediatr Neurol; 2020 Jan; 102():1-2. PubMed ID: 31481328
    [No Abstract]   [Full Text] [Related]  

  • 2. Pre-Nusinersen Hospitalization Costs of Children With Spinal Muscular Atrophy.
    Lee M; França UL; Graham RJ; McManus ML
    Pediatr Neurol; 2019 Mar; 92():3-5. PubMed ID: 30591237
    [No Abstract]   [Full Text] [Related]  

  • 3. [Gene-based therapies of spinal muscular atrophy: a piece of history of medicine].
    Braun S
    Med Sci (Paris); 2020 Feb; 36(2):141-146. PubMed ID: 32129750
    [TBL] [Abstract][Full Text] [Related]  

  • 4. More is needed to complement the available therapies of spinal muscular atrophy.
    Singh RN
    Future Med Chem; 2019 Nov; 11(22):2873-2876. PubMed ID: 31668092
    [No Abstract]   [Full Text] [Related]  

  • 5. Gene therapy for spinomuscular atrophy: a biomedical advance, a missed opportunity for more equitable drug pricing.
    Friedmann T
    Gene Ther; 2017 Sep; 24(9):503-505. PubMed ID: 28639617
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Spinal Muscular Atrophy in the Age of Gene Therapy.
    Williams L
    AACN Adv Crit Care; 2020 Mar; 31(1):86-91. PubMed ID: 32168519
    [No Abstract]   [Full Text] [Related]  

  • 7. Future avenues for therapy development for spinal muscular atrophy.
    Groen EJN
    Expert Opin Drug Discov; 2018 Oct; 13(10):899-902. PubMed ID: 30124076
    [No Abstract]   [Full Text] [Related]  

  • 8. Zolgensma - one-time gene therapy for spinal muscular atrophy.
    Med Lett Drugs Ther; 2019 Jul; 61(1577):113-114. PubMed ID: 31381549
    [No Abstract]   [Full Text] [Related]  

  • 9. Genetic therapy for spinal muscular atrophy.
    MacKenzie A
    Nat Biotechnol; 2010 Mar; 28(3):235-7. PubMed ID: 20212484
    [No Abstract]   [Full Text] [Related]  

  • 10. Advances and challenges in developing a therapy for spinal muscular atrophy.
    Anderton RS; Mastaglia FL
    Expert Rev Neurother; 2015; 15(8):895-908. PubMed ID: 26200127
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Developing therapies for spinal muscular atrophy.
    Wertz MH; Sahin M
    Ann N Y Acad Sci; 2016 Feb; 1366(1):5-19. PubMed ID: 26173388
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Trans-splicing, more than meets the eye: multifaceted therapeutics for spinal muscular atrophy.
    Butchbach ME
    Hum Gene Ther; 2011 Feb; 22(2):121-5. PubMed ID: 21288085
    [No Abstract]   [Full Text] [Related]  

  • 13. Spinal muscular atrophy: molecular pathophysiology.
    Gendron NH; MacKenzie AE
    Curr Opin Neurol; 1999 Apr; 12(2):137-42. PubMed ID: 10226744
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Guidance on Gene Replacement Therapy in Spinal Muscular Atrophy: A Canadian Perspective.
    Oskoui M; Gonorazky H; McMillan HJ; Dowling JJ; Amin R; Gagnon C; Selby K
    Can J Neurol Sci; 2022 May; 49(3):398-401. PubMed ID: 34082851
    [No Abstract]   [Full Text] [Related]  

  • 15. Gene therapy for spinal muscular atrophy: hope and caution.
    Goemans N
    Lancet Neurol; 2021 Apr; 20(4):251-252. PubMed ID: 33743228
    [No Abstract]   [Full Text] [Related]  

  • 16. Targeting splicing in spinal muscular atrophy.
    Burnett BG; Sumner CJ
    Ann Neurol; 2008 Jan; 63(1):3-6. PubMed ID: 18232015
    [No Abstract]   [Full Text] [Related]  

  • 17. Maximising returns: combining newborn screening with gene therapy for spinal muscular atrophy.
    Gillingwater TH
    J Neurol Neurosurg Psychiatry; 2021 Dec; 92(12):1252. PubMed ID: 34321342
    [No Abstract]   [Full Text] [Related]  

  • 18. Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials.
    Zanetta C; Nizzardo M; Simone C; Monguzzi E; Bresolin N; Comi GP; Corti S
    Clin Ther; 2014 Jan; 36(1):128-40. PubMed ID: 24360800
    [TBL] [Abstract][Full Text] [Related]  

  • 19. [Our initial experiences with intravenous gene therapy for spinal muscular atrophy in children].
    Mikos B; Gergely A; Balázsfi R; Bányász E; Gyömörei B; Hantos M; Czelecz J; Jakus R; Kárász H; Zilahy M; Molnár E; Paraicz É; Csohány Á; Szénási F; Vendégh L; Fogarasi A; Velkey G
    Orv Hetil; 2020 Oct; 161(42):1806-1816. PubMed ID: 33070118
    [No Abstract]   [Full Text] [Related]  

  • 20. Gene replacement therapy for spinal muscular atrophy unmasking occult hepatitis C in a pediatric patient.
    Lakhotia A; Turek G; Green J; Khan M
    Muscle Nerve; 2022 Jan; 65(1):E2-E3. PubMed ID: 34644404
    [No Abstract]   [Full Text] [Related]  

    [Next]    [New Search]
    of 9.