These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

150 related articles for article (PubMed ID: 38979994)

  • 1. A consensus survey of neurologists and clinical geneticists on spinal muscular atrophy treatment in Singapore.
    Lim JYX; Wang FS; Ling SR; Tay SKH
    Ann Acad Med Singap; 2024 Jun; 53(6):386-389. PubMed ID: 38979994
    [No Abstract]   [Full Text] [Related]  

  • 2. Cost-Effectiveness of Onasemnogene Abeparvovec Compared With Nusinersen and Risdiplam in Patients With Spinal Muscular Atrophy Type 1 in Brazil: Custo-Efetividade do Onasemnogeno Abeparvoveque (AVXS-101) em Comparação ao Nusinersena e Risdiplam em Pacientes com Atrofia Muscular Espinhal Tipo 1 no Brasil.
    Fernandes BD; D'Athayde Rodrigues F; Cardoso Cirilo HN; Borges SS; Krug BC; Probst LF; Zimmermann I
    Value Health Reg Issues; 2024 Mar; 40():108-117. PubMed ID: 38181723
    [TBL] [Abstract][Full Text] [Related]  

  • 3. 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]  

  • 4. 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]  

  • 5. [Recommendations for gene therapy of spinal muscular atrophy with onasemnogene abeparvovec-AVXS-101 : Consensus paper of the German representatives of the Society for Pediatric Neurology (GNP) and the German treatment centers with collaboration of the medical scientific advisory board of the German Society for Muscular Diseases (DGM)].
    Ziegler A; Wilichowski E; Schara U; Hahn A; Müller-Felber W; Johannsen J; von der Hagen M; von Moers A; Stoltenburg C; Saffari A; Walter MC; Husain RA; Pechmann A; Köhler C; Horber V; Schwartz O; Kirschner J
    Nervenarzt; 2020 Jun; 91(6):518-529. PubMed ID: 32394004
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Cost-Effectiveness of Technologies for the Treatment of Spinal Muscular Atrophy: A Systematic Review of Economic Studies.
    Motta-Santos A; Noronha K; Reis C; Freitas D; Carvalho L; Andrade M
    Value Health Reg Issues; 2024 Jul; 42():100985. PubMed ID: 38669792
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Spinal Muscular Atrophy.
    Nicolau S; Waldrop MA; Connolly AM; Mendell JR
    Semin Pediatr Neurol; 2021 Apr; 37():100878. PubMed ID: 33892848
    [TBL] [Abstract][Full Text] [Related]  

  • 8. How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison.
    Ribero VA; Daigl M; Martí Y; Gorni K; Evans R; Scott DA; Mahajan A; Abrams KR; Hawkins N
    J Comp Eff Res; 2022 Apr; 11(5):347-370. PubMed ID: 35040693
    [No Abstract]   [Full Text] [Related]  

  • 9. [Spinal muscular atrophy].
    Martin P; Horber V; Park J; Kronlage C; Grimm A
    Nervenarzt; 2022 Feb; 93(2):191-200. PubMed ID: 35037967
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Challenges and opportunities in spinal muscular atrophy therapeutics.
    Yeo CJJ; Tizzano EF; Darras BT
    Lancet Neurol; 2024 Feb; 23(2):205-218. PubMed ID: 38267192
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Spinal Muscular Atrophy Treatments, Newborn Screening, and the Creation of a Neurogenetics Urgency.
    Butterfield RJ
    Semin Pediatr Neurol; 2021 Jul; 38():100899. PubMed ID: 34183144
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Switching therapies: safety profile of Onasemnogene abeparvovec-xioi in a SMA1 patient previously treated with Risdiplam.
    Tosi M; Catteruccia M; Cherchi C; Mizzoni I; D'Amico A
    Acta Myol; 2022; 41(3):117-120. PubMed ID: 36349185
    [TBL] [Abstract][Full Text] [Related]  

  • 13. 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]  

  • 14. Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study.
    Weiß C; Ziegler A; Becker LL; Johannsen J; Brennenstuhl H; Schreiber G; Flotats-Bastardas M; Stoltenburg C; Hartmann H; Illsinger S; Denecke J; Pechmann A; Müller-Felber W; Vill K; Blaschek A; Smitka M; van der Stam L; Weiss K; Winter B; Goldhahn K; Plecko B; Horber V; Bernert G; Husain RA; Rauscher C; Trollmann R; Garbade SF; Hahn A; von der Hagen M; Kaindl AM
    Lancet Child Adolesc Health; 2022 Jan; 6(1):17-27. PubMed ID: 34756190
    [TBL] [Abstract][Full Text] [Related]  

  • 15. 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]  

  • 16. Safety Concerns with Nusinersen, Risdiplam, and Onasemnogene Abeparvovec in Spinal Muscular Atrophy: A Real-World Pharmacovigilance Study.
    Zhuang W; Lu M; Wu Y; Chen Z; Wang M; Wang X; Guan S; Lin W
    Clin Drug Investig; 2023 Dec; 43(12):949-962. PubMed ID: 37995087
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Spinal Muscular Atrophy in the Treatment Era.
    Waldrop MA; Elsheikh BH
    Neurol Clin; 2020 Aug; 38(3):505-518. PubMed ID: 32703464
    [TBL] [Abstract][Full Text] [Related]  

  • 18. [Update on spinal muscular atrophy treatment].
    Erazo Torricelli R
    Medicina (B Aires); 2022 Aug; 82 Suppl 3():76-81. PubMed ID: 36054863
    [TBL] [Abstract][Full Text] [Related]  

  • 19. 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]  

  • 20. Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovec.
    Leon-Astudillo C; Wagner M; Salabarria SM; Lammers J; Berthy J; Zingariello CD; Byrne BJ; Smith BK
    Sleep Med; 2023 Jan; 101():234-237. PubMed ID: 36442421
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 8.