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1. Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec

2. Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA)

3. Correction to: Two‑year efficacy and safety of risdiplam in patients with type 2 or non‑ambulant type 3 spinal muscular atrophy (SMA)

4. CGG repeat expansion in LRP12 in amyotrophic lateral sclerosis

5. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

6. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

8. Precision cancer genome testing needs proficiency testing involving all stakeholders

9. 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

11. Lifesaving Treatments for Spinal Muscular Atrophy: Global Access and Availability (P5-4.003)

12. Phenotypes of SMA patients retaining SMN1 with intragenic mutation

13. Newborn screening programs for spinal muscular atrophy worldwide: Where we stand and where to go

14. Survey of patients with spinal muscular atrophy on the island of Shikoku, Japan

16. Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

17. Sunfish parts 1 and 2: 4-year efficacy and safety data of risdiplam in types 2 and 3 spinal muscular atrophy (SMA)

18. Cybernic treatment with wearable cyborg Hybrid Assistive Limb (HAL) improves ambulatory function in patients with slowly progressive rare neuromuscular diseases: a multicentre, randomised, controlled crossover trial for efficacy and safety (NCY-3001)

19. Genetic abnormalities in a large cohort of Coffin–Siris syndrome patients

22. Combination disease‐modifying treatment in spinal muscular atrophy: A proposed classification

24. Spinal muscular atrophy carriers with two SMN1 copies

25. Effective Valproic Acid Treatment in Motor Function Is Caused by Possible Mechanism of Elevated Survival Motor Neuron Protein Related With Splicing Factor Gene Expression in Spinal Muscular Atrophy

40. Intragenic mutations in SMN1 may contribute more significantly to clinical severity than SMN2 copy numbers in some spinal muscular atrophy (SMA) patients

41. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

42. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

44. Onasemnogene Abeparvovec for Presymptomatic Infants with Spinal Muscular Atrophy and Two Copies of SMN2 (S39.004)

45. Effectiveness and Safety of Onasemnogene Abeparvovec in Older Patients with Spinal Muscular Atrophy (SMA): Real-World Outcomes from the RESTORE Registry (S39.002)

46. The RESTORE Registry: Real-World Assessments of Interventions and Long-Term Outcomes in Patients with Spinal Muscular Atrophy (SMA) (P15-5.002)

48. Onasemnogene Abeparvovec in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with Three Copies of SMN2 (P15-5.001)

49. The RESTORE Registry: Comparative Outcomes in Patients with Spinal Muscular Atrophy (SMA) in the United States Identified by Newborn Screening (NBS) or Clinical Diagnosis (P15-5.003)

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