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1. Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study

2. Benefits of Early Versus Late Initiation of Intravenous Immunoglobulin in the Treatment of Patients With Anti–3‐Hydroxy‐3‐Methylglutaryl‐Coenzyme A Reductase Immune‐Mediated Necrotizing Myopathy.

3. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

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

8. CIDP mimics: a case series

15. Diagnostic utility of exome sequencing in the evaluation of neuromuscular disorders

17. Determining Best or Inferior Drug(s) Using an Adaptive Platform for Cryptogenic Sensory Polyneuropathy

21. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

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

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

27. CIDP mimics: a case series

31. Contributors

38. Selective serotonin reuptake inhibitors ameliorate MEGF10 myopathy

42. Clinical Sequencing Exploratory Research Consortium: Accelerating Evidence-Based Practice of Genomic Medicine

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

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