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HOME > Therapeutic areas > Rare Diseases

The European Union defines a disease as rare if it affects no more than 5 in 10,000 people in the EU. Up to 8,000 rare diseases exist worldwide. Around four million people live with a rare disease in Germany alone; in the EU the number is estimated at 30 million.

In most cases, rare diseases present a highly complex clinical picture. One thing that they have in common is a typically chronic progression. Sufferers are frequently affected by disabilities and/or have a limited life expectancy. Symptoms generally appear in childhood. Around 80% of rare diseases have genetic origins or are in part due to genetic damage, and they are seldom curable.

At Chiesi, a separate business unit is dedicated to rare diseases, Chiesi Global Rare Diseases.

These include: 

  • ADA-SCID - Adenosine Deaminase Deficiency Severe Combined Immunodeficiency
  • Alpha-Mannosidosis
  • Cystinosis
  • Epidermolysis bullosa
  • LHON - Leber's Hereditary Optic Neuropathy
  • Lipodystrophy
  • Morbus Fabry
  • Thalassemia