108 results on '"Sproule, Douglas M"'
Search Results
2. Correction to: Validation of a novel western blot assay to monitor patterns and levels of alpha dystroglycan in skeletal muscle of patients with limb girdle muscular dystrophies
3. Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue
4. 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
5. Response to: Alfred Sandrock, Wildon Farwell. Letter to the Editor, Comparisons Between Separately Conducted Clinical Trials: Letter to the Editor Regarding Dabbous O, Maru B, Jansen JP, Lorenzi M, Cloutier M, Guérin A, et al. Adv Ther (2019) 36(5):1164-76. doi:10.1007/s12325-019-00923-8
6. Stroke-Like Episodes in Mitochondrial Myopathy, Encephalopathy, Lactic Acidosis, and Stroke-Like Episodes (MELAS)
7. High Healthcare Resource Use in Hospitalized Patients with a Diagnosis of Spinal Muscular Atrophy Type 1 (SMA1): Retrospective Analysis of the Kids’ Inpatient Database (KID)
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
9. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
10. Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy
11. Giant axonal neuropathy: An updated perspective on its pathology and pathogenesis
12. THE MOTOR NEURON RESPONSE TO SMN1 DEFICIENCY IN SPINAL MUSCULAR ATROPHY
13. An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)
14. Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1 (SMA1): Phase 3 US Study (STR1VE) Update (1828)
15. Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy in Presymptomatic Spinal Muscular Atrophy: SPR1NT Study Update (2384)
16. Gene-Replacement Therapy in Spinal Muscular Atrophy Type 1: Long-Term Follow-Up From the Onasemnogene Abeparvovec-xioi Phase 1/2a Clinical Trial (1808)
17. One-Time Intrathecal (IT) Administration of AVXS-101 IT Gene-Replacement Therapy for Spinal Muscular Atrophy: Phase 1 Study (STRONG) (2493)
18. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort
19. Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update
20. Study Design of STR1VE-EU, a Phase 3 Trial of AVXS-101 Gene-Replacement Therapy (GRT) in Patients With Spinal Muscular Atrophy Type 1 (SMA1) in Europe
21. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy
22. Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2 Clinical Trial
23. 066 Avxs-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) update
24. 014 AVXS-101 gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): study update
25. AVXS-101 Gene-Replacement Therapy (GRT) in Presymptomatic Spinal Muscular Atrophy (SMA): Study Update (P1.6-057)
26. AVXS-101 Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Phase 1 Clinical Trial (S25.006)
27. Number Needed to Treat (NNT) in Spinal Muscular Atrophy Type 1 (SMA1) with AVXS-101 Relative to Nusinersen (P1.6-056)
28. AVXS-101 Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update (P1.6-058)
29. The Value of AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy Type 1: Improved Survival, Pulmonary and Nutritional Support, and Motor Function with Decreased Hospitalization (P1.6-060)
30. Early Diagnosis and Speed to Effect in Spinal Muscular Atrophy Type 1 (SMA1) (S25.002)
31. Event-Free Survival and Motor Milestone Achievement Following AVXS-101 and Nusinersen Interventions Contrasted to Natural History for Type I Spinal Muscular Atrophy Patients (S25.005)
32. Phase 1 Study of Intrathecal Administration of AVXS-101 Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 2 (SMA2) (STRONG) (P1.6-059)
33. Diagnostic précoce et rapidité de l’effet sur l’amyotrophie spinale infantile de type 1 (ASI-1)
34. Comparaison de la survie, de la fonction motrice et des étapes de développement moteur de l’AVXS-101 et du nusinersen dans le traitement des nourrissons atteints d’amyotrophie spinale de type 1 (ASI-1)
35. Fardeau économique de l’amyotrophie spinale de type 1 (ASI-1) d’apparition chez le nourrisson : analyse rétrospective d’une base de données
36. Essai clinique portant sur la thérapie de remplacement d’un gène par AVXS-101 dans le traitement de l’amyotrophie spinale (ASI) pré-symptomatique : conception de l’étude de phase 3 et données démographiques lors de la visite de référence initiale
37. Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients
38. Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy
39. High Healthcare Resource Use in Hospitalized Patients with a Diagnosis of Spinal Muscular Atrophy Type 1 (SMA1): Retrospective Analysis of the Kids’ Inpatient Database (KID)
40. AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in SMA Type 1: Continued Independence from Nutritional and Ventilatory Support in Patients Dosed Early in Disease Progression (S29.003)
41. AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in SMA Type 1: Continued Event Free Survival and Achievement of Developmental Milestones (S29.001)
42. AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in SMA Type 1: Patients Treated Early with the Proposed Therapeutic Dose Were Able to Sit Unassisted at a Younger Age (S29.002)
43. AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones
44. AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Interim data demonstrates improvements in supportive care use
45. CSF delivery of AAV9-mediated gene therapy for SMA, a lethal neuromuscular disease in children: a dose-response study in mice and nonhuman primates (P3.133)
46. AVXS-101 Phase 1 Gene Therapy Clinical Trial in SMA Type 1: Correlation between CHOP-INTEND and Motor Milestone achievements (S13.002)
47. AVXS-101 Phase 1 Gene Therapy Clinical Trial in SMA Type 1: Experience with pre-existing anti-AAV9 antibody in the SMA1 population (S13.001)
48. Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2 Clinical Trial.
49. Study Design of STR1VE-EU, a Phase 3 Trial of AVXS-101 Gene-Replacement Therapy (GRT) in Patients With Spinal Muscular Atrophy Type 1 (SMA1) in Europe.
50. Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy.
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