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1. Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial

4. P281 Quality of life in adults with dysferlinopathy: international clinical outcome study of dysferlinopathy

5. Biallelic variants in COQ7 cause distal hereditary motor neuropathy with upper motor neuron signs

6. Expanding the muscle imaging spectrum in dysferlinopathy: description of an outlier population from the classical MRI pattern

8. Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy

10. FP.35 Myostatin concentration is unreliable as a biomarker of disease progression in dysferlinopathy

11. Randomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease

12. Randomized phase 2 study of ACE‐083, a muscle‐promoting agent, in facioscapulohumeral muscular dystrophy

13. A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week Study of the Efficacy and Safety of Losmapimod in Subjects with FSHD: ReDUX4 (S23.007)

14. Whole Body MRI Quantitative muscle analysis to evaluate Efficacy of Losmapimod in a Phase 2 Placebo-Controlled Study in Subjects with FSHD (ReDUX4) (S23.009)

15. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach

16. Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

19. Investigating Late-Onset Pompe Prevalence in Neuromuscular Medicine Academic Practices

20. LGMD

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

24. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease

26. Clinical utility of anti‐cytosolic 5’‐nucleotidase 1A antibody in idiopathic inflammatory myopathies

28. Intensive Teenage Activity Is Associated With Greater Muscle Hyperintensity on T1W Magnetic Resonance Imaging in Adults With Dysferlinopathy

30. Contributors

32. Contributors

34. FSHD / OPMD / MYOTONIC DYSTROPHY

35. FROM THE SPINAL CORD TO THE MUSCLE

36. Epidemiological evidence for a hereditary contribution to myasthenia gravis: a retrospective cohort study of patients from North America

37. Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

38. Loss- or Gain-of-Function Mutations in ACOX1 Cause Axonal Loss via Different Mechanisms

41. Results of a Phase 2 Double-Blind Placebo-Controlled Study of a Local Muscle Therapeutic, ACE-083, in Subjects with Charcot-Marie-Tooth (CMT) Disease (1514)

42. Design of a Phase 2, Randomized, Double-Blind, Placebo-Controlled, 24-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD): ReDUX4 (1592)

43. Myositis associated anti-NT5C1A autoantibody in clinical practice

46. Contributing Authors

49. Selection design phase II trial of high dosages of tamoxifen and creatine in amyotrophic lateral sclerosis

50. P.183Functional progression in dysferlinopathy: results of a 3-year natural history study

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