233 results on '"Duong, Tina"'
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2. Upper limb function changes over 12 months in untreated SMA II and III individuals: an item-level analysis using the Revised Upper Limb Module
3. Performance of upper limb entry item to predict forced vital capacity in dysferlin-deficient limb girdle muscular dystrophy
4. Changes in abilities over the initial 12 months of nusinersen treatment for type II SMA
5. Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study
6. Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study
7. Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
8. Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0.
9. Major Adverse Dystrophinopathy Events (MADE) score as marker of cumulative morbidity and risk for mortality in boys with Duchenne muscular dystrophy
10. Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
11. Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2–3 trial
12. Genetic modifiers of upper limb function in Duchenne muscular dystrophy
13. Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial
14. Emerging therapies for Duchenne muscular dystrophy
15. Long‐term natural history in type II and III spinal muscular atrophy: a 4‐year international study on the Hammersmith Functional Motor Scale Expanded.
16. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial
17. Revised upper limb module in type II and III spinal muscular atrophy: 24-month changes
18. Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy
19. Advances in the Therapy of Spinal Muscular Atrophy
20. Age related treatment effect in type II Spinal Muscular Atrophy pediatric patients treated with nusinersen
21. Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study
22. Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study.
23. Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: An international study.
24. Gain and loss of abilities in type II SMA: A 12-month natural history study
25. Correction to: Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers’ perspective: a two-part study
26. Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers’ perspective: a two-part study
27. Development of an academic disease registry for spinal muscular atrophy
28. Patients' Perceptions of Nusinersen Effects According to Their Responder Status.
29. Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3.
30. Nusinersen Treatment in Adults with Spinal Muscular Atrophy
31. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study
32. Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study.
33. Determining An Appropriate Cardiopulmonary Exercise Testing Protocol For Individuals With Neuromuscular Disease: 2369 Board #288 May 28 3:00 PM - 4:30 PM
34. Longitudinal changes in clinical outcome measures in COL6-related dystrophies and LAMA2-related dystrophies
35. Consensus-based care recommendations for congenital and childhood-onset myotonic dystrophy type 1
36. Assessment of disease progression in dysferlinopathy: A 1-year cohort study
37. Assessing the Assisted Six-Minute Cycling Test as a Measure of Endurance in Non-Ambulatory Patients with Spinal Muscular Atrophy (SMA).
38. Cerebrospinal Fluid Proteomic Changes after Nusinersen in Patients with Spinal Muscular Atrophy.
39. Validity and reliability of smartphone magnetometer-based goniometer evaluation of shoulder abduction – A pilot study
40. Results of a two-year pilot study of clinical outcome measures in collagen VI- and laminin alpha2-related congenital muscular dystrophies
41. Consensus-based care recommendations for adults with myotonic dystrophy type 1
42. Quantitative Evaluation of Lower Extremity Joint Contractures in Spinal Muscular Atrophy: Implications for Motor Function
43. Electrical impedance myography in individuals with collagen 6 and laminin α‐2 congenital muscular dystrophy: a cross‐sectional and 2‐year analysis
44. Development of an International SMA Bulbar Assessment for Inter-professional Administration.
45. Effects Of Different Exercise Regimen On The Disease Phenotype In Mdx Mouse Model Of DMD: 2697 Board #217 June 2 11: 00 AM - 12: 30 PM
46. In Vitro Cytotoxicity of Antiresorptive and Antiangiogenic Compounds on Oral Tissues Contributing to MRONJ: Systematic Review.
47. Bulbar function in spinal muscular atrophy (SMA): State of art and new challenges. 21st July 2023, Rome, Italy
48. Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes.
49. Genetic modifiers of ambulation in the cooperative international Neuromuscular research group Duchenne natural history study
50. 269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy – What have we learned? 9–11 December 2022, Hoofddorp, The Netherlands
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