142 results on '"Voit, T."'
Search Results
2. DMD – ANIMAL MODELS
3. DMD – BIOMARKERS & OUTCOME MEASURES
4. DMD – THERAPY
5. P.204Full-body behaviour analytics reveals DMD disease state within the first few steps of the 6-minute-walk test
6. P.203Towards high-resolution clinical digital biomarkers for Duchenne muscular dystrophy
7. P.144A cross sectional and longitudinal miRNA profiling study identified a set of novel free-circulating and exosomal miRNAs dysregulated in plasma from Duchenne muscular dystrophy patients
8. P.264SYROS study – long-term reduction in rate of respiratory function decline in patients with Duchenne muscular dystrophy treated with idebenone
9. P.261Consistent long-term effect of idebenone in reducing respiratory function decline in advanced patients with Duchenne muscular dystrophy
10. P.149The MYODA operational seamless clinical trial design phase I to III: a new approach for rare diseases to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of BIO101 (MAS activator) in paediatric patients with a genetically confirmed diagnosis of Duchenne muscular dystrophy
11. P.205Daily life digital biomarkers for longitudinal monitoring of Duchenne muscular dystrophy with wearable sensors
12. EP.87MYODA clinical program: composite score for assessing the efficacy of BIO101 (MAS activator) in ambulatory and non-ambulatory Duchenne boys
13. Respiratory and upper limb function as outcome measures in ambulant and non-ambulant subjects with Duchenne muscular dystrophy: A prospective multicentre study
14. NEW THERAPEUTIC APPROACHES AND THEIR READOUT
15. DMD CLINICAL THERAPIES II
16. DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY
17. Downregulation of miR-29 and miR-23 in urine of Duchenne muscular dystrophy patients
18. Myostatin is a reliable biomarker for monitoring drug response in DMD
19. Myostatin expression in neuromuscular diseases
20. A new AAV10-mediated gene therapy for SOD1 -linked ALS
21. Myostatin expression levels in neuromuscular diseases participates in anti-myostatin clinical failure
22. Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant patients: implications for clinical trials
23. Reversible endogenous downregulation of myostatin pathway in wasting neuromuscular diseases explains challenges of anti-myostatin therapeutic approaches
24. Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy
25. Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: implications for clinical trials
26. Myostatin inhibition for neuromuscular disorders: defining the good candidate
27. Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: Implications for clinical trials
28. Movement monitoring at home and during study visits identifies sources of variability in 6MWT performance in Duchenne muscular dystrophy
29. Cardiac arrhythmia and late-onset muscle weakness caused by a myofibrillar myopathy with unusual histopathological features due to a novel missense mutation in FLNC
30. Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy
31. Longitudinal results of magneto-inertial motion analysis in Duchenne muscular dystrophy ambulant patients
32. Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy
33. Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy
34. Natural history of respiratory function changes in patients with Duchenne muscular dystrophy not using glucocorticoid steroids
35. A European prospective study of the natural history of patients with type 2 and 3 spinal muscular atrophy
36. Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: Implications for clinical trials
37. Childhood Pompe disease: Clinical spectrum and genotype in 31 children
38. Adeno-associated virus vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne muscular dystrophy (DMD)
39. Natural history and functional status of patients with myotubular myopathy enrolled in a prospective and longitudinal study
40. Serum and urine proteomic profiling reveals biomarkers suitable for monitoring the outcome of therapeutic interventions in muscular dystrophies
41. Elastic properties of skeletal muscle and subcutaneous tissues in Duchenne muscular dystrophy by magnetic resonance elastography (MRE): A feasibility study
42. G.P.114
43. T.I.1
44. G.P.93
45. T.P.13
46. G.O.23
47. G.P.39
48. G.P.116
49. G.P.92
50. G.P.115
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