42 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. 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)
7. Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1
8. The absence of curly hair is associated with a milder phenotype in Giant Axonal Neuropathy
9. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
10. Adiposity is increased among high-functioning, non-ambulatory patients with spinal muscular atrophy
11. Increased fat mass and high incidence of overweight despite low body mass index in patients with spinal muscular atrophy
12. Giant axonal neuropathy: An updated perspective on its pathology and pathogenesis
13. THE MOTOR NEURON RESPONSE TO SMN1 DEFICIENCY IN SPINAL MUSCULAR ATROPHY
14. Scoliosis Surgery in Children With Neuromuscular Disease: Findings From the US National Inpatient Sample, 1997 to 2003
15. 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).
16. Mitochondrial Encephalopathy, Lactic Acidosis, and Strokelike Episodes: Basic Concepts, Clinical Phenotype, and Therapeutic Management of MELAS Syndrome
17. Wolff-Parkinson-White Syndrome in Patients With MELAS
18. Monitoring of Cerebral Vasodilatory Capacity With Transcranial Doppler Carbon Dioxide Inhalation in Patients With Severe Carotid Artery Disease
19. Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients.
20. Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2 Clinical Trial.
21. 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.
22. Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy.
23. Measuring Disease Progression in Giant Axonal Neuropathy: Implications for Clinical Trial Design.
24. Stroke-Like Episodes in Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-Like Episodes (MELAS).
25. Observational study of spinal muscular atrophy type I and implications for clinical trials.
26. Diabetic Ketoacidosis in an Adult Patient With Spinal Muscular Atrophy Type II: Further Evidence of Extraneural Pathology Due to Survival Motor Neuron 1 Mutation?
27. Weakness and Fatigue in Diverse Neuromuscular Diseases.
28. Independent Mobility After Early Introduction of a Power Wheelchair in Spinal Muscular Atrophy.
29. SMA-MAP: A Plasma Protein Panel for Spinal Muscular Atrophy.
30. Prospective cohort study of spinal muscular atrophy types 2 and 3.
31. Age at Disease Onset Predicts Likelihood and Rapidity of Growth Failure Among Infants and Young Children With Spinal Muscular Atrophy Types 1 and 2.
32. Spinal Muscular Atrophy Type III: Trying to Understand Subtle Functional Change Over Time—A Case Report.
33. Thigh Muscle Volume Measured by Magnetic Resonance Imaging Is Stable Over a 6-Month Interval in Spinal Muscular Atrophy.
34. Muscle Volume Estimation by Magnetic Resonance Imaging in Spinal Muscular Atrophy.
35. Bioelectrical Impedance Analysis Can Be a Useful Screen for Excess Adiposity in Spinal Muscular Atrophy.
36. Therapeutic developments in spinal muscular atrophy.
37. AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones.
38. AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Interim data demonstrates improvements in supportive care use.
39. REPLY.
40. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy.
41. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort.
42. Reply: To PMID 23893312.
Catalog
Books, media, physical & digital resources
Discovery Service for Jio Institute Digital Library
For full access to our library's resources, please sign in.