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 *

1733 related articles for article (PubMed ID: 29091557)

  • 1. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
    Mendell JR; Al-Zaidy S; Shell R; Arnold WD; Rodino-Klapac LR; Prior TW; Lowes L; Alfano L; Berry K; Church K; Kissel JT; Nagendran S; L'Italien J; Sproule DM; Wells C; Cardenas JA; Heitzer MD; Kaspar A; Corcoran S; Braun L; Likhite S; Miranda C; Meyer K; Foust KD; Burghes AHM; Kaspar BK
    N Engl J Med; 2017 Nov; 377(18):1713-1722. PubMed ID: 29091557
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.
    Darras BT; Masson R; Mazurkiewicz-Bełdzińska M; Rose K; Xiong H; Zanoteli E; Baranello G; Bruno C; Vlodavets D; Wang Y; El-Khairi M; Gerber M; Gorni K; Khwaja O; Kletzl H; Scalco RS; Fontoura P; Servais L;
    N Engl J Med; 2021 Jul; 385(5):427-435. PubMed ID: 34320287
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy.
    Al-Zaidy S; Pickard AS; Kotha K; Alfano LN; Lowes L; Paul G; Church K; Lehman K; Sproule DM; Dabbous O; Maru B; Berry K; Arnold WD; Kissel JT; Mendell JR; Shell R
    Pediatr Pulmonol; 2019 Feb; 54(2):179-185. PubMed ID: 30548438
    [TBL] [Abstract][Full Text] [Related]  

  • 4. 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]  

  • 5. 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]  

  • 6. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.
    Finkel RS; Chiriboga CA; Vajsar J; Day JW; Montes J; De Vivo DC; Yamashita M; Rigo F; Hung G; Schneider E; Norris DA; Xia S; Bennett CF; Bishop KM
    Lancet; 2016 Dec; 388(10063):3017-3026. PubMed ID: 27939059
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 8. 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]  

  • 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. 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]  

  • 11. Risdiplam in Type 1 Spinal Muscular Atrophy.
    Baranello G; Darras BT; Day JW; Deconinck N; Klein A; Masson R; Mercuri E; Rose K; El-Khairi M; Gerber M; Gorni K; Khwaja O; Kletzl H; Scalco RS; Seabrook T; Fontoura P; Servais L;
    N Engl J Med; 2021 Mar; 384(10):915-923. PubMed ID: 33626251
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.
    Finkel RS; Mercuri E; Darras BT; Connolly AM; Kuntz NL; Kirschner J; Chiriboga CA; Saito K; Servais L; Tizzano E; Topaloglu H; Tulinius M; Montes J; Glanzman AM; Bishop K; Zhong ZJ; Gheuens S; Bennett CF; Schneider E; Farwell W; De Vivo DC;
    N Engl J Med; 2017 Nov; 377(18):1723-1732. PubMed ID: 29091570
    [TBL] [Abstract][Full Text] [Related]  

  • 13. 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]  

  • 14. 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]  

  • 15. Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes.
    Waldrop MA; Karingada C; Storey MA; Powers B; Iammarino MA; Miller NF; Alfano LN; Noritz G; Rossman I; Ginsberg M; Mosher KA; Broomall E; Goldstein J; Bass N; Lowes LP; Tsao CY; Mendell JR; Connolly AM
    Pediatrics; 2020 Sep; 146(3):. PubMed ID: 32843442
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Nusinersen in patients older than 7 months with spinal muscular atrophy type 1: A cohort study.
    Aragon-Gawinska K; Seferian AM; Daron A; Gargaun E; Vuillerot C; Cances C; Ropars J; Chouchane M; Cuppen I; Hughes I; Illingworth M; Marini-Bettolo C; Rambaud J; Taytard J; Annoussamy M; Scoto M; Gidaro T; Servais L
    Neurology; 2018 Oct; 91(14):e1312-e1318. PubMed ID: 30158155
    [TBL] [Abstract][Full Text] [Related]  

  • 17. An observational study of functional abilities in infants, children, and adults with type 1 SMA.
    Pane M; Palermo C; Messina S; Sansone VA; Bruno C; Catteruccia M; Sframeli M; Albamonte E; Pedemonte M; D'Amico A; Brigati G; de Sanctis R; Coratti G; Lucibello S; Bertini E; Vita G; Danilo Tiziano F; Mercuri E;
    Neurology; 2018 Aug; 91(8):e696-e703. PubMed ID: 30045959
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN.
    Hinderer C; Katz N; Buza EL; Dyer C; Goode T; Bell P; Richman LK; Wilson JM
    Hum Gene Ther; 2018 Mar; 29(3):285-298. PubMed ID: 29378426
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Spinal muscular atrophy: survival pattern and functional status.
    Chung BH; Wong VC; Ip P
    Pediatrics; 2004 Nov; 114(5):e548-53. PubMed ID: 15492357
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Clinical Outcomes in Patients with Spinal Muscular Atrophy Type 1 Treated with Nusinersen.
    de Holanda Mendonça R; Jorge Polido G; Ciro M; Jorge Fontoura Solla D; Conti Reed U; Zanoteli E
    J Neuromuscul Dis; 2021; 8(2):217-224. PubMed ID: 33459657
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 87.