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 *

169 related articles for article (PubMed ID: 38160362)

  • 1. Parental Experiences with Newborn Screening and Gene Replacement Therapy for Spinal Muscular Atrophy.
    Meyer AP; Connolly AM; Vannatta K; Hacker N; Hatfield A; Decipeda A; Parker P; Willoughby A; Waldrop MA
    J Neuromuscul Dis; 2024; 11(1):129-142. PubMed ID: 38160362
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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]  

  • 3. Willingness to Pay for a Newborn Screening Test for Spinal Muscular Atrophy.
    Lin PJ; Yeh WS; Neumann PJ
    Pediatr Neurol; 2017 Jan; 66():69-75. PubMed ID: 27769729
    [TBL] [Abstract][Full Text] [Related]  

  • 4. 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]  

  • 5. Real-Life Outcome After Gene Replacement Therapy for Spinal Muscular Atrophy: A Multicenter Experience.
    Tokatly Latzer I; Sagi L; Lavi R; Aharoni S; Bistritzer J; Noyman I; Ginsburg M; Lev-Or A; Katzenellenbogen S; Nevo Y; Fattal-Valevski A
    Pediatr Neurol; 2023 Jul; 144():60-68. PubMed ID: 37149951
    [TBL] [Abstract][Full Text] [Related]  

  • 6. 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]  

  • 7. 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]  

  • 8. 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]  

  • 9. 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]  

  • 10. Spinal Muscular Atrophy: The Past, Present, and Future of Diagnosis and Treatment.
    Nishio H; Niba ETE; Saito T; Okamoto K; Takeshima Y; Awano H
    Int J Mol Sci; 2023 Jul; 24(15):. PubMed ID: 37569314
    [TBL] [Abstract][Full Text] [Related]  

  • 11. 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]  

  • 12. Parent Perceptions in Choosing Treatment for Infants With Spinal Muscular Atrophy Diagnosed Through Newborn Screening.
    Deng S; Lee BH; Ciafaloni E
    J Child Neurol; 2022 Jan; 37(1):43-49. PubMed ID: 34753336
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Evolving approaches to prenatal genetic counseling for Spinal Muscular Atrophy in the new treatment era.
    Zettler B; Estrella E; Liaquat K; Lichten L
    J Genet Couns; 2022 Jun; 31(3):803-814. PubMed ID: 35037741
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Sleep disordered breathing in infants identified through newborn screening with spinal muscular atrophy.
    Chiang J; Xiao L; Nigro E; St-Laurent A; Weinstock L; Law E; Janevski J; Kuyntjes S; Cithiravel N; Tran T; Wolter NE; Gonorazky H; Amin R;
    Sleep Med; 2023 Nov; 111():161-169. PubMed ID: 37778092
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Newborn screening programs for spinal muscular atrophy worldwide: Where we stand and where to go.
    Dangouloff T; Vrščaj E; Servais L; Osredkar D;
    Neuromuscul Disord; 2021 Jun; 31(6):574-582. PubMed ID: 33985857
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Newborn genetic screening for spinal muscular atrophy in the UK: The views of the general population.
    Boardman FK; Sadler C; Young PJ
    Mol Genet Genomic Med; 2018 Jan; 6(1):99-108. PubMed ID: 29169204
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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]  

  • 18. Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma).
    Ogbonmide T; Rathore R; Rangrej SB; Hutchinson S; Lewis M; Ojilere S; Carvalho V; Kelly I
    Cureus; 2023 Mar; 15(3):e36197. PubMed ID: 37065340
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Progress in spinal muscular atrophy research.
    Wurster C; Petri S
    Curr Opin Neurol; 2022 Oct; 35(5):693-698. PubMed ID: 35942665
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy.
    Kichula EA; Proud CM; Farrar MA; Kwon JM; Saito K; Desguerre I; McMillan HJ
    Muscle Nerve; 2021 Oct; 64(4):413-427. PubMed ID: 34196026
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 9.