BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

338 related articles for article (PubMed ID: 33677142)

  • 41. Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1.
    Dabbous O; Maru B; Jansen JP; Lorenzi M; Cloutier M; Guérin A; Pivneva I; Wu EQ; Arjunji R; Feltner D; Sproule DM
    Adv Ther; 2019 May; 36(5):1164-1176. PubMed ID: 30879249
    [TBL] [Abstract][Full Text] [Related]  

  • 42. Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series.
    Chand DH; Zaidman C; Arya K; Millner R; Farrar MA; Mackie FE; Goedeker NL; Dharnidharka VR; Dandamudi R; Reyna SP
    J Pediatr; 2021 Apr; 231():265-268. PubMed ID: 33259859
    [TBL] [Abstract][Full Text] [Related]  

  • 43. The respiratory impact of novel therapies for spinal muscular atrophy.
    Paul GR; Gushue C; Kotha K; Shell R
    Pediatr Pulmonol; 2021 Apr; 56(4):721-728. PubMed ID: 33098622
    [TBL] [Abstract][Full Text] [Related]  

  • 44. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy.
    Lowes LP; Alfano LN; Arnold WD; Shell R; Prior TW; McColly M; Lehman KJ; Church K; Sproule DM; Nagendran S; Menier M; Feltner DE; Wells C; Kissel JT; Al-Zaidy S; Mendell J
    Pediatr Neurol; 2019 Sep; 98():39-45. PubMed ID: 31277975
    [TBL] [Abstract][Full Text] [Related]  

  • 45. Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis.
    Pascual-Morena C; Cavero-Redondo I; Lucerón-Lucas-Torres M; Martínez-García I; Rodríguez-Gutiérrez E; Martínez-Vizcaíno V
    Hum Gene Ther; 2023 Feb; 34(3-4):129-138. PubMed ID: 36136906
    [TBL] [Abstract][Full Text] [Related]  

  • 46. Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment.
    McGrattan KE; Shell RD; Hurst-Davis R; Young SD; O'Brien E; Lavrov A; Wallach S; LaMarca N; Reyna SP; Darras BT
    J Neuromuscul Dis; 2023; 10(4):531-540. PubMed ID: 37092232
    [TBL] [Abstract][Full Text] [Related]  

  • 47. Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom.
    Gowda V; Atherton M; Murugan A; Servais L; Sheehan J; Standing E; Manzur A; Scoto M; Baranello G; Munot P; McCullagh G; Willis T; Tirupathi S; Horrocks I; Dhawan A; Eyre M; Vanegas M; Fernandez-Garcia MA; Wolfe A; Pinches L; Illingworth M; Main M; Abbott L; Smith H; Milton E; D'Urso S; Vijayakumar K; Marco SS; Warner S; Reading E; Douglas I; Muntoni F; Ong M; Majumdar A; Hughes I; Jungbluth H; Wraige E
    Lancet Reg Health Eur; 2024 Feb; 37():100817. PubMed ID: 38169987
    [TBL] [Abstract][Full Text] [Related]  

  • 48. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort.
    Al-Zaidy SA; Kolb SJ; Lowes L; Alfano LN; Shell R; Church KR; Nagendran S; Sproule DM; Feltner DE; Wells C; Ogrinc F; Menier M; L'Italien J; Arnold WD; Kissel JT; Kaspar BK; Mendell JR
    J Neuromuscul Dis; 2019; 6(3):307-317. PubMed ID: 31381526
    [TBL] [Abstract][Full Text] [Related]  

  • 49. Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis.
    Fernandes BD; Krug BC; Rodrigues FD; Cirilo HNC; Borges SS; Schwartz IVD; Probst LF; Zimmermann I
    PLoS One; 2024; 19(5):e0302860. PubMed ID: 38713659
    [TBL] [Abstract][Full Text] [Related]  

  • 50. Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy.
    René CA; Parks RJ
    Pharmaceutics; 2023 Jun; 15(6):. PubMed ID: 37376212
    [TBL] [Abstract][Full Text] [Related]  

  • 51. Time is muscle: A recommendation for early treatment for preterm infants with spinal muscular atrophy.
    Lee BH; Waldrop MA; Connolly AM; Ciafaloni E
    Muscle Nerve; 2021 Aug; 64(2):153-155. PubMed ID: 33959970
    [TBL] [Abstract][Full Text] [Related]  

  • 52. Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey.
    Toro W; Yang M; Georgieva M; Anderson A; LaMarca N; Patel A; Akbarnejad H; Dabbous O
    Adv Ther; 2023 Dec; 40(12):5315-5337. PubMed ID: 37776479
    [TBL] [Abstract][Full Text] [Related]  

  • 53. Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment.
    Monnette A; Chen E; Hong D; Bazzano A; Dixon S; Arnold WD; Shi L
    Orphanet J Rare Dis; 2021 Jan; 16(1):36. PubMed ID: 33472673
    [TBL] [Abstract][Full Text] [Related]  

  • 54. Spinal Muscular Atrophy.
    Nance JR
    Continuum (Minneap Minn); 2020 Oct; 26(5):1348-1368. PubMed ID: 33003005
    [TBL] [Abstract][Full Text] [Related]  

  • 55. A breakthrough effect of gene replacement therapy on respiratory outcomes in children with spinal muscular atrophy.
    AlNaimi A; Hamad SG; Mohamed RBA; Ben-Omran T; Ibrahim K; Osman MFE; Abu-Hasan M
    Pediatr Pulmonol; 2023 Apr; 58(4):1004-1011. PubMed ID: 36533697
    [TBL] [Abstract][Full Text] [Related]  

  • 56. Newborn screening for spinal muscular atrophy with disease-modifying therapies: a cost-effectiveness analysis.
    Shih ST; Farrar MA; Wiley V; Chambers G
    J Neurol Neurosurg Psychiatry; 2021 Dec; 92(12):1296-1304. PubMed ID: 34321343
    [TBL] [Abstract][Full Text] [Related]  

  • 57. Nusinersen treatment of spinal muscular atrophy - a systematic review.
    Albrechtsen SS; Born AP; Boesen MS
    Dan Med J; 2020 Aug; 67(9):. PubMed ID: 32800069
    [TBL] [Abstract][Full Text] [Related]  

  • 58. Onasemnogene Abeparvovec: First Global Approval.
    Hoy SM
    Drugs; 2019 Jul; 79(11):1255-1262. PubMed ID: 31270752
    [TBL] [Abstract][Full Text] [Related]  

  • 59. Recombinant Adeno-Associated Virus Serotype 9 Gene Therapy in Spinal Muscular Atrophy.
    Kotulska K; Fattal-Valevski A; Haberlova J
    Front Neurol; 2021; 12():726468. PubMed ID: 34721262
    [TBL] [Abstract][Full Text] [Related]  

  • 60. Will the US$5 million onasemnogene abeparvosec treatment for spinal muscular atrophy represent 'value for money' for the NHS? A rapid inquiry into suggestions that it may be cost-effective.
    Connock M; Andronis L; Auguste P; Dussart C; Armoiry X
    Expert Opin Biol Ther; 2020 Jul; 20(7):823-827. PubMed ID: 32434404
    [TBL] [Abstract][Full Text] [Related]  

    [Previous]   [Next]    [New Search]
    of 17.