Orphan Medicines

In addition to our broad therapeutic experience, GMAS has developed expertise in issues relating to the market access of orphan medicines.

As well as having conducted research on policy issues relating to the funding of treatments for rare diseases, we have specific disease knowledge in the following areas:

• Fabry disease
• Multiple myeloma
• Lambert-Eaton Myasthenic Syndrome
• Myeloproliferative disorders:
  • Myelodysplastic syndromes
  • Essential thrombocythemia
  • Polycythemia rubra vera
• Haemophilia, Von Willebrand’s disease and other bleeding disorders
• Thalassaemia
• Idiopathic thrombocytopenic purpura
• Primary arterial hypertension
• Multifocal motor neuropathy
• Chronic Inflammatory demyelinating polyneuropathy
• Cystic fibrosis
• Amyotrophic lateral sclerosis
• Barrett’s oesophagus
• Hereditary angiodema.