BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

476 related articles for article (PubMed ID: 34721262)

  • 21. Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.
    Servais L; Day JW; De Vivo DC; Kirschner J; Mercuri E; Muntoni F; Proud CM; Shieh PB; Tizzano EF; Quijano-Roy S; Desguerre I; Saito K; Faulkner E; Benguerba KM; Raju D; LaMarca N; Sun R; Anderson FA; Finkel RS
    J Neuromuscul Dis; 2024; 11(2):425-442. PubMed ID: 38250783
    [TBL] [Abstract][Full Text] [Related]  

  • 22. Pharmacotherapy for Spinal Muscular Atrophy in Babies and Children: A Review of Approved and Experimental Therapies.
    Chiriboga CA
    Paediatr Drugs; 2022 Nov; 24(6):585-602. PubMed ID: 36028610
    [TBL] [Abstract][Full Text] [Related]  

  • 23. Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec.
    Day JW; Finkel RS; Mercuri E; Swoboda KJ; Menier M; van Olden R; Tauscher-Wisniewski S; Mendell JR
    Mol Ther Methods Clin Dev; 2021 Jun; 21():76-82. PubMed ID: 33768131
    [TBL] [Abstract][Full Text] [Related]  

  • 24. [Treatment of Spinal Muscular Atrophy].
    Ishiyama A
    Brain Nerve; 2023 May; 75(5):507-510. PubMed ID: 37194521
    [TBL] [Abstract][Full Text] [Related]  

  • 25. Health Care Resource Utilization and Costs for Patients with Spinal Muscular Atrophy: Findings from a Retrospective US Claims Database Analysis.
    Toro W; Yang M; Georgieva M; Song W; Patel A; Jiang AX; Zhao A; LaMarca N; Dabbous O
    Adv Ther; 2023 Oct; 40(10):4589-4605. PubMed ID: 37587305
    [TBL] [Abstract][Full Text] [Related]  

  • 26. Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio.
    Waldrop MA; Chagat S; Storey M; Meyer A; Iammarino M; Reash N; Alfano L; Lowes L; Noritz G; Prochoroff A; Rossman I; Ginsberg M; Mosher K; Broomall E; Bass N; Gushue C; Kotha K; Paul G; Shell R; Tsao CY; Mendell JR; Connolly AM
    Neuromuscul Disord; 2024 Jan; 34():41-48. PubMed ID: 38142474
    [TBL] [Abstract][Full Text] [Related]  

  • 27. New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges.
    Messina S; Sframeli M
    J Clin Med; 2020 Jul; 9(7):. PubMed ID: 32668756
    [TBL] [Abstract][Full Text] [Related]  

  • 28. Bridging the Gap: Gene Therapy in a Patient With Spinal Muscular Atrophy Type 1.
    Costamagna G; Govoni A; Wise A; Corti S
    Neurology; 2022 Nov; 99(21):952-956. PubMed ID: 36319109
    [TBL] [Abstract][Full Text] [Related]  

  • 29. Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue.
    Thomsen G; Burghes AHM; Hsieh C; Do J; Chu BTT; Perry S; Barkho B; Kaufmann P; Sproule DM; Feltner DE; Chung WK; McGovern VL; Hevner RF; Conces M; Pierson CR; Scoto M; Muntoni F; Mendell JR; Foust KD
    Nat Med; 2021 Oct; 27(10):1701-1711. PubMed ID: 34608334
    [TBL] [Abstract][Full Text] [Related]  

  • 30. Selecting disease-modifying medications in 5q spinal muscular atrophy.
    Cartwright MS; Upadhya S
    Muscle Nerve; 2021 Oct; 64(4):404-412. PubMed ID: 34231920
    [TBL] [Abstract][Full Text] [Related]  

  • 31. Long term peripheral AAV9-SMN gene therapy promotes survival in a mouse model of spinal muscular atrophy.
    Reilly A; Yaworski R; Beauvais A; Schneider BL; Kothary R
    Hum Mol Genet; 2024 Feb; 33(6):510-519. PubMed ID: 38073249
    [TBL] [Abstract][Full Text] [Related]  

  • 32. Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec.
    Retson L; Tiwari N; Vaughn J; Bernes S; Adelson PD; Mansfield K; Libertini S; Kuzmiski B; Alecu I; Gabriel R; Mangum R
    Mol Ther; 2023 Oct; 31(10):2991-2998. PubMed ID: 37598295
    [TBL] [Abstract][Full Text] [Related]  

  • 33. Matching-adjusted indirect treatment comparison of onasemnogene abeparvovec and nusinersen for the treatment of symptomatic patients with spinal muscular atrophy type 1.
    Bischof M; Lorenzi M; Lee J; Druyts E; Balijepalli C; Dabbous O
    Curr Med Res Opin; 2021 Oct; 37(10):1719-1730. PubMed ID: 34236007
    [TBL] [Abstract][Full Text] [Related]  

  • 34. Onasemnogene abeparvovec-xioi: a gene replacement strategy for the treatment of infants diagnosed with spinal muscular atrophy.
    Schwartz M; Likhite S; Meyer K
    Drugs Today (Barc); 2021 Jun; 57(6):387-399. PubMed ID: 34151905
    [TBL] [Abstract][Full Text] [Related]  

  • 35. Combination Therapy with Nusinersen and Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy Type I.
    Mirea A; Shelby ES; Axente M; Badina M; Padure L; Leanca M; Dima V; Sporea C
    J Clin Med; 2021 Nov; 10(23):. PubMed ID: 34884240
    [TBL] [Abstract][Full Text] [Related]  

  • 36. Safety Monitoring of Gene Therapy for Spinal Muscular Atrophy with Onasemnogene Abeparvovec -A Single Centre Experience.
    Friese J; Geitmann S; Holzwarth D; Müller N; Sassen R; Baur U; Adler K; Kirschner J
    J Neuromuscul Dis; 2021; 8(2):209-216. PubMed ID: 33427694
    [TBL] [Abstract][Full Text] [Related]  

  • 37. European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy.
    Kirschner J; Butoianu N; Goemans N; Haberlova J; Kostera-Pruszczyk A; Mercuri E; van der Pol WL; Quijano-Roy S; Sejersen T; Tizzano EF; Ziegler A; Servais L; Muntoni F
    Eur J Paediatr Neurol; 2020 Sep; 28():38-43. PubMed ID: 32763124
    [TBL] [Abstract][Full Text] [Related]  

  • 38. Gene therapy for spinal muscular atrophy is considerably effective when administered as early as possible after birth.
    Sawada T; Kido J; Yae Y; Yuge K; Nomura K; Okada K; Fujiyama N; Ozasa S; Nakamura K
    Mol Genet Metab Rep; 2023 Jun; 35():100973. PubMed ID: 37091744
    [TBL] [Abstract][Full Text] [Related]  

  • 39. Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy.
    Day JW; Mendell JR; Burghes AHM; van Olden RW; Adhikary RR; Dilly KW
    Mol Ther Methods Clin Dev; 2023 Dec; 31():101117. PubMed ID: 37822718
    [TBL] [Abstract][Full Text] [Related]  

  • 40. Current and emerging therapies for Duchenne muscular dystrophy and spinal muscular atrophy.
    Iftikhar M; Frey J; Shohan MJ; Malek S; Mousa SA
    Pharmacol Ther; 2021 Apr; 220():107719. PubMed ID: 33130193
    [TBL] [Abstract][Full Text] [Related]  

    [Previous]   [Next]    [New Search]
    of 24.