The European Union defines a disease as rare if not more than 5 in 10,000 people in the EU are affected by this disease. Worldwide, there are up to 8,000 “rare diseases”. In Austria, around 400,000 people suffer from a rare disease; throughout the EU, the number is an estimated 30 million people. In most cases, rare diseases are characterised by a very complex clinical picture. Around 80% of rare diseases are genetic in origin or can be partially traced back to genetic causes. Many of these diseases have their onset in childhood. The progression of these diseases is often chronic and means a long life of suffering for the patients.

Active ingredients

Cysteamine

Trade name: Procysbi®

For interested parties from Austria: For more detailed information, please refer to the published patient information leaflet (EMA - European Medicines agency ) or consult your doctor or pharmacist.

Velmanase alfa

Trade name: Lamzede®

For interested parties from Austria: For more detailed information, please refer to the published patient information leaflet (EMA - European Medicines agency ) or consult your doctor or pharmacist.

Deferipron

Trade name: Ferriprox®

For interested parties from Austria: For more detailed information, please refer to the published patient information leaflet (EMA - European Medicines agency ) or consult your doctor or pharmacist.

Idebenon

Trade name: Raxone®

For interested parties from Austria: For more detailed information, please refer to the published patient information leaflet (EMA - European Medicines agency ) or consult your doctor or pharmacist.

Pegunigalsidase Alpha

Trade name: Elfabrio®

For interested parties from Austria: For more detailed information, please refer to the published patient information leaflet (EMA - European Medicines agency ) or consult your doctor or pharmacist.