195 results on '"Sproule, Douglas M"'
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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. Stroke-Like Episodes in Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-Like Episodes (MELAS)
10. The absence of curly hair is associated with a milder phenotype in Giant Axonal Neuropathy
11. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
12. Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy
13. Adiposity is increased among high-functioning, non-ambulatory patients with spinal muscular atrophy
14. Increased fat mass and high incidence of overweight despite low body mass index in patients with spinal muscular atrophy
15. Giant axonal neuropathy: An updated perspective on its pathology and pathogenesis
16. THE MOTOR NEURON RESPONSE TO SMN1 DEFICIENCY IN SPINAL MUSCULAR ATROPHY
17. 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)
18. Stroke-Like Episodes in Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-Like Episodes (MELAS)
19. Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1 (SMA1): Phase 3 US Study (STR1VE) Update (1828)
20. Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy in Presymptomatic Spinal Muscular Atrophy: SPR1NT Study Update (2384)
21. Gene-Replacement Therapy in Spinal Muscular Atrophy Type 1: Long-Term Follow-Up From the Onasemnogene Abeparvovec-xioi Phase 1/2a Clinical Trial (1808)
22. One-Time Intrathecal (IT) Administration of AVXS-101 IT Gene-Replacement Therapy for Spinal Muscular Atrophy: Phase 1 Study (STRONG) (2493)
23. Scoliosis Surgery in Children With Neuromuscular Disease: Findings From the US National Inpatient Sample, 1997 to 2003
24. Mitochondrial Encephalopathy, Lactic Acidosis, and Strokelike Episodes: Basic Concepts, Clinical Phenotype, and Therapeutic Management of MELAS Syndrome
25. Wolff-Parkinson-White Syndrome in Patients With MELAS
26. Monitoring of Cerebral Vasodilatory Capacity With Transcranial Doppler Carbon Dioxide Inhalation in Patients With Severe Carotid Artery Disease
27. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort
28. Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update
29. 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
30. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy
31. Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2 Clinical Trial
32. 066 Avxs-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) update
33. 014 AVXS-101 gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): study update
34. AVXS-101 Gene-Replacement Therapy (GRT) in Presymptomatic Spinal Muscular Atrophy (SMA): Study Update (P1.6-057)
35. 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)
36. Number Needed to Treat (NNT) in Spinal Muscular Atrophy Type 1 (SMA1) with AVXS-101 Relative to Nusinersen (P1.6-056)
37. AVXS-101 Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update (P1.6-058)
38. 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)
39. Early Diagnosis and Speed to Effect in Spinal Muscular Atrophy Type 1 (SMA1) (S25.002)
40. 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)
41. Phase 1 Study of Intrathecal Administration of AVXS-101 Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 2 (SMA2) (STRONG) (P1.6-059)
42. Diagnostic précoce et rapidité de l’effet sur l’amyotrophie spinale infantile de type 1 (ASI-1)
43. 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)
44. Fardeau économique de l’amyotrophie spinale de type 1 (ASI-1) d’apparition chez le nourrisson : analyse rétrospective d’une base de données
45. 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
46. Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients
47. Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy
48. 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)
49. 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)
50. AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in SMA Type 1: Continued Event Free Survival and Achievement of Developmental Milestones (S29.001)
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