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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

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

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

32. Caregivers’ Expectations on Possible Functional Changes following Disease-Modifying Treatment in Type II and III Spinal Muscular Atrophy: A Comparative Study

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

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

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

50. Quantitative Evaluation of Lower Extremity Joint Contractures in Spinal Muscular Atrophy: Implications for Motor Function

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