325 results on '"Coratti, Giorgia"'
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2. Prognostic factors for tube feeding in type I SMA patients treated with disease-modifying therapies: a cohort study
3. Early neurological signs in infants identified through neonatal screening for SMA: do they predict outcome?
4. Assessing Prevalence and Characteristics of Oro-bulbar Involvement in Children and Adults with SMA Type 2 and 3 Using a Multimodal Approach
5. Changes in abilities over the initial 12 months of nusinersen treatment for type II SMA
6. Gain and loss of upper limb abilities in Duchenne muscular dystrophy patients: A 24-month study
7. Profile of cognitive abilities in spinal muscular atrophy type II and III: what is the role of motor impairment?
8. Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0.
9. Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies
10. Neurological assessment of newborns with spinal muscular atrophy identified through neonatal screening
11. Assessing floppy infants: a new module
12. Bulbar function in spinal muscular atrophy (SMA): State of art and new challenges. 21st July 2023, Rome, Italy
13. Body mass index in type 2 spinal muscular atrophy: a longitudinal study
14. Long term follow-up of scoliosis progression in type II SMA patients
15. Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial
16. Revised upper limb module in type II and III spinal muscular atrophy: 24-month changes
17. 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
18. Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: An international study.
19. Communicative development inventory in type 1 and presymptomatic infants with spinal muscular atrophy: a cohort study
20. Can the CHOP-INTEND be used as An Outcome Measure in the First Months of Age? Implications for Clinical Trials and Real World Data
21. Age related treatment effect in type II Spinal Muscular Atrophy pediatric patients treated with nusinersen
22. The nonsense mutation stop+4 model correlates with motor changes in Duchenne muscular dystrophy
23. The Spinal Muscular Atrophy Health Index: Italian validation of a disease-specific outcome measure
24. Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3.
25. Respiratory Needs in Patients with Type 1 Spinal Muscular Atrophy Treated with Nusinersen
26. Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis
27. Development of an International SMA Bulbar Assessment for Inter-professional Administration
28. Early treatment of type II SMA slows rate of progression of scoliosis.
29. Development of an International SMA Bulbar Assessment for Inter-professional Administration
30. Respiratory Trajectories in Type 2 and 3 Spinal Muscular Atrophy in the iSMAC Cohort Study
31. 6MWT can identify type 3 SMA patients with neuromuscular junction dysfunction
32. Caregivers’ Expectations on Possible Functional Changes following Disease-Modifying Treatment in Type II and III Spinal Muscular Atrophy: A Comparative Study
33. Measuring Fatigue and Fatigability in Spinal Muscular Atrophy (SMA): Challenges and Opportunities
34. Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies
35. Longitudinal Analysis of PUL 2.0 Domains in Ambulant and Non-Ambulant Duchenne Muscular Dystrophy Patients: How do they Change in Relation to Functional Ability?
36. Sleep disorders in spinal muscular atrophy
37. Longitudinal natural history of type I spinal muscular atrophy: a critical review
38. Clinical Phenotype of Pediatric and Adult Patients With Spinal Muscular Atrophy With Four SMN2 Copies: Are They Really All Stable?
39. Developmental milestones in type I spinal muscular atrophy
40. 2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA Participants
41. A Longitudinal Follow-Up Study of Intellectual Function in Duchenne Muscular Dystrophy over Age: Is It Really Stable?
42. Upper Limb Changes in DMD Patients Amenable to Skipping Exons 44, 45, 51 and 53: A 24-Month Study
43. Type I spinal muscular atrophy patients treated with nusinersen: 4-year follow-up of motor, respiratory and bulbar function
44. Longitudinal Analysis of PUL 2.0 Domains in Ambulant and Non-Ambulant Duchenne Muscular Dystrophy Patients: How do they Change in Relation to Functional Ability?
45. Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy
46. Long-term progression in type II spinal muscular atrophy: A retrospective observational study
47. Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial
48. Language Development in Preschool Duchenne Muscular Dystrophy Boys
49. An observational study of functional abilities in infants, children, and adults with type 1 SMA
50. Quantitative Evaluation of Lower Extremity Joint Contractures in Spinal Muscular Atrophy: Implications for Motor Function
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