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4. P.134a Phase 1/2a trial of delandistrogene moxeparvovec in patients with DMD: 4-year update

6. DMD - TREATMENT

7. S11 Long-term follow-up of the phase 1 START trial of onasemnogene abeparvovec gene therapy in spinal muscular atrophy type 1

9. DMD – THERAPY

10. SMA – THERAPY

11. OUTCOME MEASURES

13. Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1: Pulmonary and Ventilatory Findings from the Pivotal Phase 3 US Study (STR1VE)

16. S61 Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): preliminary pulmonary and ventilatory findings from the phase 3 study (STR1VE)

17. Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): Long-term follow-up from the onasemnogene abeparvovec phase 1/2A clinical trial

19. P.351Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the onasemnogene abeparvovec phase 1/2a clinical trial

20. AVXS-101 Gene Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update

23. SMA THERAPIES I

24. SMA THERAPIES I

25. SMA THERAPIES I

26. PSY16 - HEALTH OUTCOME IMPROVEMENTS IN SPINAL MUSCULAR ATROPHY TYPE 1 PATIENTS WITH AVXS-101 GENE REPLACEMENT THERAPY

28. Nusinersen versus sham control in infantile-onset spinal muscular atrophy

29. AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: decreased need of ventilatory and nutritional support at End-of-Study

30. AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: end-of-Study event free survival and achievement of developmental milestones

31. AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: patients treated early with the proposed therapeutic dose were able to sit unassisted at a younger age

32. Ab initio studies of the vibrational spectra and structure of phosphorus halides. I. Selection of basis functions and the spectra of PCl3.

39. Your list's 'worth': adding it up

44. Back to the future

47. Theoretical basis for general mixed object handling equations based on mechanical work required.

48. Conserved Receptor-binding Domains of Lake Victoria Marburgvirus and Zaire Ebolavirus Bind a Common Receptor.

49. Psychosocial factors and musculoskeletal disorders in the construction industry: a systematic review.

50. Workplace bullying: a systematic review of risk factors and outcomes.

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