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1. SNUPN deficiency causes a recessive muscular dystrophy due to RNA mis-splicing and ECM dysregulation

2. Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy.

3. Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio

4. Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study.

5. Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians

6. Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy

7. Findings from the Longitudinal CINRG Becker Natural History Study

8. Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in Duchenne Muscular Dystrophy (DMD): Meeting Report from the 3rd International Muscle-Bone Interactions Meeting 7th and 14th November 2022

9. Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

10. Childhood amyotrophic lateral sclerosis caused by excess sphingolipid synthesis

13. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

14. Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study

16. Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!

17. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

20. Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

23. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy

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

28. Contributors

29. Natural history of infantile‐onset spinal muscular atrophy

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

34. Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network

35. Mutations in sphingosine-1-phosphate lyase cause nephrosis with ichthyosis and adrenal insufficiency

39. Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

42. Clinical and genetic characterization of manifesting carriers of DMD mutations

43. Validity and Reliability of the Neuromuscular Gross Motor Outcome

44. Clinical and Molecular Spectrum Associated with COL6A3 c.7447A>G p.(Lys2483Glu) Variant: Elucidating its Role in Collagen VI-related Myopathies

45. Correction to: Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study

46. Findings from the Longitudinal CINRG Becker Natural History Study

47. Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in Duchenne Muscular Dystrophy (DMD): Meeting Report from the 3rd International Muscle-Bone Interactions Meeting 7th and 14th November 2022

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