BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

153 related articles for article (PubMed ID: 37553206)

  • 1. Effect on maximal mouth opening in children with spinal muscular atrophy treated with onasemnogene abeparvovec.
    Beri N; Kapoor L; Parashar D; Mundada V
    Arch Dis Child; 2023 Oct; 108(10):866-867. PubMed ID: 37553206
    [No Abstract]   [Full Text] [Related]  

  • 2. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.
    Mercuri E; Muntoni F; Baranello G; Masson R; Boespflug-Tanguy O; Bruno C; Corti S; Daron A; Deconinck N; Servais L; Straub V; Ouyang H; Chand D; Tauscher-Wisniewski S; Mendonca N; Lavrov A;
    Lancet Neurol; 2021 Oct; 20(10):832-841. PubMed ID: 34536405
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Safety of Onasemnogene Abeparvovec for Patients With Spinal Muscular Atrophy 8.5 kg or Heavier in a Global Managed Access Program.
    Chand DH; Mitchell S; Sun R; LaMarca N; Reyna SP; Sutter T
    Pediatr Neurol; 2022 Jul; 132():27-32. PubMed ID: 35605311
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series.
    Gaillard J; Gu AR; Neil Knierbein EE
    J Pediatr; 2023 Sep; 260():113493. PubMed ID: 37211209
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Onasemnogene abeparvovec preserves bulbar function in infants with presymptomatic spinal muscular atrophy: a post-hoc analysis of the SPR1NT trial.
    Shell RD; McGrattan KE; Hurst-Davis R; Young SD; Baranello G; Lavrov A; O'Brien E; Wallach S; LaMarca N; Reyna SP; Darras BT
    Neuromuscul Disord; 2023 Aug; 33(8):670-676. PubMed ID: 37455203
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study.
    Bitetti I; Lanzara V; Margiotta G; Varone A
    Gene Ther; 2023 Aug; 30(7-8):592-597. PubMed ID: 35606491
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Gene transfer therapy in children with spinal muscular atrophy: A single-center experience with a cohort of 25 children.
    Chencheri N; Alexander G; Nugud A; Majadas E; Salim H; Prudhomme K; DeJager N; Janardhanan VS; Elbashir H
    Muscle Nerve; 2023 Sep; 68(3):269-277. PubMed ID: 37392188
    [TBL] [Abstract][Full Text] [Related]  

  • 8. 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]  

  • 9. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.
    Day JW; Finkel RS; Chiriboga CA; Connolly AM; Crawford TO; Darras BT; Iannaccone ST; Kuntz NL; Peña LDM; Shieh PB; Smith EC; Kwon JM; Zaidman CM; Schultz M; Feltner DE; Tauscher-Wisniewski S; Ouyang H; Chand DH; Sproule DM; Macek TA; Mendell JR
    Lancet Neurol; 2021 Apr; 20(4):284-293. PubMed ID: 33743238
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Safety and efficacy of gene therapy with onasemnogene abeparvovec in the treatment of spinal muscular atrophy: A systematic review and meta-analysis.
    Yang D; Ruan Y; Chen Y
    J Paediatr Child Health; 2023 Mar; 59(3):431-438. PubMed ID: 36722610
    [TBL] [Abstract][Full Text] [Related]  

  • 11. 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]  

  • 12. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1.
    Al-Zaidy SA; Mendell JR
    Pediatr Neurol; 2019 Nov; 100():3-11. PubMed ID: 31371124
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Polysomnography findings and respiratory muscle function in infants with early onset spinal muscular atrophy after gene replacement as monotherapy: A prospective study.
    Barrois R; Griffon L; Barnerias C; Gitiaux C; Desguerre I; Fauroux B; Khirani S
    Sleep Med; 2024 Jul; 119():335-341. PubMed ID: 38749258
    [TBL] [Abstract][Full Text] [Related]  

  • 14. 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]  

  • 15. Onasemnogene abeparvovec for the treatment of spinal muscular atrophy.
    McMillan HJ; Proud CM; Farrar MA; Alexander IE; Muntoni F; Servais L
    Expert Opin Biol Ther; 2022 Sep; 22(9):1075-1090. PubMed ID: 35437095
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial.
    Strauss KA; Farrar MA; Muntoni F; Saito K; Mendell JR; Servais L; McMillan HJ; Finkel RS; Swoboda KJ; Kwon JM; Zaidman CM; Chiriboga CA; Iannaccone ST; Krueger JM; Parsons JA; Shieh PB; Kavanagh S; Wigderson M; Tauscher-Wisniewski S; McGill BE; Macek TA
    Nat Med; 2022 Jul; 28(7):1390-1397. PubMed ID: 35715567
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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]  

  • 18. [Pharmacological and clinical profile of Onasemnogene Aveparvovec, the first gene therapy for spinal muscular atrophy (SMA)].
    Atsumi A; Yoneda T; Tsuchida K; Kagawa Y; Tominaga S; Kawase K; Kikuchi N
    Nihon Yakurigaku Zasshi; 2022; 157(1):53-61. PubMed ID: 34980814
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Acute Benign Myositis Following Onasemnogene Abeparvovec Therapy in Type 1 Spinal Muscular Atrophy.
    Dosi C; Tozzo A; Masson R
    Pediatr Neurol; 2022 Jun; 131():23-24. PubMed ID: 35462108
    [No Abstract]   [Full Text] [Related]  

  • 20. 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]  

    [Next]    [New Search]
    of 8.