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3. Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial

4. Zilucoplan in immune-mediated necrotising myopathy: a phase 2, randomised, double-blind, placebo-controlled, multicentre trial

5. Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

6. Long-term effect of thymectomy plus prednisone versus prednisone alone in patients with non-thymomatous myasthenia gravis: 2-year extension of the MGTX randomised trial

7. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial

8. Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease

10. Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study

11. A Phonetic Complexity-Based Approach for Intelligibility and Articulatory Precision Testing: A Preliminary Study on Talkers with Amyotrophic Lateral Sclerosis

12. Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial

13. Phase 2 Trial of Rituximab in Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: The BeatMG Study

14. Longitudinal course of neurofilament light chain levels in amyotrophic lateral sclerosis—insights from a completed randomized controlled trial with rasagiline.

16. Genome-wide Analyses Identify KIF5A as a Novel ALS Gene

17. Lingual-Alveolar Contact Pressure during Speech in Amyotrophic Lateral Sclerosis: Preliminary Findings

18. NEO1/NEO-EXT studies: Long-term muscle quantitative magnetic resonance imaging and functional efficacy in adults with late-onset Pompe disease (LOPD) on avalglucosidase alfa treatment

19. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

20. Minimal manifestation status and prednisone withdrawal in the MGTX trial

23. Episodic Ataxia Type 1: Natural History and Effect on Quality of Life.

32. 2017 European League Against Rheumatism/American College of Rheumatology classification criteria for adult and juvenile idiopathic inflammatory myopathies and their major subgroups

33. Using a Bayesian model of the joint distribution of pain and time on medication to decide on pain medication for neuropathy.

34. Phase 2 trial in acetylcholine receptor antibody‐positive myasthenia gravis of transition from intravenous to subcutaneous immunoglobulin: The MGSCIg study.

37. A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial

38. Activity for Diabetic Polyneuropathy (ADAPT): study design and protocol for a 2-site randomized controlled trial

42. Editorial by concerned physicians: Unintended effect of the orphan drug act on the potential cost of 3,4-diaminopyridine

43. Rare variants in SQSTM1 and VCP genes and risk of sporadic inclusion body myositis

45. A Genome-Wide Association Study of Myasthenia Gravis

46. Efficacy and Safety of Bimagrumab in Sporadic Inclusion Body Myositis: Long-term Extension of RESILIENT

47. A secondary analysis of PAIN‐CONTRoLS: Pain's impact on sleep, fatigue, and activities of daily living.

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