504 results on '"Guglieri, M."'
Search Results
2. Correction: Developing a Natural History Model for Duchenne Muscular Dystrophy
3. The impact of testosterone therapy on quality of life in adolescents with Duchenne muscular dystrophy
4. P156 Muscle biopsy findings in a large cohort of patients affected by valosin containing protein disease: preliminary analysis of the international multicentric VCP study
5. P242 Understanding North Star Ambulatory Assessment total scores and their implications for standards of care
6. P147 Six-year long-term safety and efficacy of Golodirsen in patients with DMD vs mutation-matched external controls
7. P268 Decoding the transcriptome of Duchenne muscular dystrophy to the single nuclei level reveals clinical-genetic correlations
8. P98 Quality of life in adults with neuromuscular conditions and the association with diagnosis and mobility status
9. VP38 Changes to glucocorticosteroid prescribing patterns in Duchenne muscular dystrophy in the UK over the last decade
10. P191 Exercise prescription for patients with neuromuscular diseases: Lessons learned from real-world data on exercise exposure
11. P240 Improving care for Duchenne muscular dystrophy: examples of the impact of collaborative working in DMD Care UK – cardiac, respiratory, psychosocial and emergency care
12. P239 The DMD Hub: a UK network enabling trials and recruitment to studies in Duchenne muscular dystrophy via the central recruitment database
13. P39 The effect of corticosteroid treatment on pulmonary function in adults with Duchenne muscular dystrophy
14. P246 Non-glucocorticoid related comorbidities in adults with Duchenne muscular dystrophy
15. P149 Delayed pulmonary progression in Golodirsen-treated patients with Duchenne muscular dystrophy vs mutation-matched external controls
16. P193 TREAT-NMD global registry network: an insight into a global neuromuscular patient dataset
17. P243 Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids
18. Characteristics and survival of patients with advanced heart failure treated with left ventricular assist device (LVAD) as an effective bridge to heart transplantation vs. destination therapy through a French national retrospective cohort
19. Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne muscular dystrophy
20. P.24 Consensus and collaboration approach to better defining and implementing harmonised standards of care across a healthcare system: Examples from DMD care
21. P.213 TREAT-NMD myotonic dystrophy global registry network: An international collaboration in myotonic dystrophy type 2
22. O.11 Identification of a novel heterozygous DYSF variant in a large family with a dominantly-inherited dysferlinopathy
23. P.133 Daily regimens of prednisone, deflazacort and vamorolone improve motor function similarly in patients with Duchenne muscular dystrophy
24. P.122 Comparative safety and efficacy of different corticosteroid regimens in boys with Duchenne muscular dystrophy: results of a randomized controlled trial
25. FP.03 The spine fracture burden in boys with DMD treated with the novel dissociative steroid vamorolone versus deflazacort and prednisone
26. FP.26 Early effect of corticosteroids on functional outcomes in young patients with Duchenne muscular dystrophy within the first 18 months of treatment
27. FP.09 Analysis of muscle MRI of a large cohort of chronic motor neuropathy/neuronopathy patients reveals characteristic features useful for diagnosis
28. VP.24 T Cell-mediated immune response to dystrophin in Duchenne muscular dystrophy - A natural history study
29. P.212 TREAT-NMD Myotonic dystrophy (DM) Global Registry Network: An update in 2022
30. P.211 TREAT-NMD myotonic dystrophy Global Registry Network: Providing data in congenital myotonic dystrophy to support FDA regulatory decision making
31. P.71 Vamorolone has less impact than daily prednisone or deflazacort on height and body mass index in patients with Duchenne muscular dystrophy (DMD)
32. FP.30 TREAT-NMD FSHD Global Registry Network: A collaboration of neuromuscular and FSHD patient registries
33. FP.27 Results of a double-blind cross-over trial of vamorolone in DMD: A safer alternative to corticosteroids
34. P.14 Analysis of the longitudinal CINRG Becker natural history study dataset
35. P.125 DMD Hub: A UK network enabling trials in Duchenne muscular dystrophy
36. FP.35 Myostatin concentration is unreliable as a biomarker of disease progression in dysferlinopathy
37. Axial involvement as a prominent feature in SMPX-related distal myopathy
38. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach
39. Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy : A Randomized Clinical Trial
40. Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients
41. Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta‐analysis of disease progression rates in recent multicenter clinical trials
42. Variability of advice and education for steroid sick day dosing in Duchenne Muscular Dystrophy: Results of a UK patient survey
43. REGISTRIES AND CARE OF NMD
44. SMA – OUTCOME MEASURES AND REGISTRIES
45. LGMD
46. DMD – CLINICAL CARE
47. DMD – CLINICAL CARE
48. DMD/BMD – OUTCOME MEASURES
49. DMD - TREATMENT
50. DMD/BMD – OUTCOME MEASURES
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