The use of intravenous immunoglobulin (IVIg) for the treatment of immune-meditated peripheral neuropathies (Guillain-Barre syndrome, chronic inflammatory demyelinating polyneuropathy, multifocal motor neuropathy), myasthenia gravis and inflammatory myopathies. This has largely replaced plasma exchange and has made a major impact on the management and clinical outcomes in these conditions. The downside is that IVIg therapy is very expensive and is not as freely available in less well-developed countries.
Cytokine-based therapies such as tumor necrosis factor a (TNF a) inhibitors for the treatment of immune-mediated inflammatory myopathies - this is still an emerging therapy which is undergoing elevation in clinical trials.
Monoclonal antibodies: rituximab which induces B-cell depletion - for the treatment of certain types of immune-mediated inflammatory neuropathies and myopathies; alemtuzumab which induces T-cell depletion and is currently being evaluated for the treatment of certain T-cell mediated disorders such as inclusion body myositis and polymyositis.
Enzyme replacement therapy with alglucosidase alfa (Myozyme) for Pompe's disease (adult acid maltase deficiency).
Exon-skipping approaches using antisense oligonucleotides in Duchenne Muscular Dystrophy (DMD), and possibly other hereditary neuromuscular disorders.
Stem-cell therapy for inherited neuromuscular disorders.
Gene therapy for inherited neuromuscular disorders.
Up-regulation of fetal isoforms in hereditary disorders (e.g.,myophosphorylase deficiency) among others.
