Orphan drugs to treat myasthenia gravis

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Introduction: Acquired myasthenia gravis (MG) is a rare disease and according to the EU Register of designated Orphan Medicinal Products only two drugs are registered as orphan drugs for MG while no orphan drug for MG has been approved by the US Food and Drug Administration. The aim of this review is to discuss the role and mechanism of action of the recognized orphan drugs for MG and of other therapies potentially useful for MG treatment, which should be considered as orphan drugs. Areas covered: In the context of an updated overview of MG pathogenesis, the authors discuss current MG treatments available in clinical practice, orphan drugs for MG according to the EU Orphan Drugs Registry and emerging therapies, among which are therapies exclusively developed for MG. Expert opinion: New effective drugs for MG are needed to reduce side effects, mainly those related to steroids or to chronic immunosuppression, and to manage patients not responsive to common treatments. Hence, in recent years drugs potentially targeting selective pathogenetic steps of MG have been developed, although the complexity of the immune system makes their application very challenging. To this purpose a great effort at multiple levels, including basic research, clinical practice and health politics, will be necessary.

Original languageEnglish
Pages (from-to)373-384
Number of pages12
JournalExpert Opinion on Orphan Drugs
Issue number5
Publication statusPublished - 2013


  • Cellular therapies
  • Emerging therapies
  • Monoclonal antibodies
  • Myasthenia gravis
  • Orphan drugs
  • Treatment

ASJC Scopus subject areas

  • Pharmacology (medical)
  • Health Policy
  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)

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