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Rare diseases

Each patient has the right to the best available treatment. This applies especially to the patients suffering from the so-called rare diseases. These are most often genetic metabolic diseases, but they can occur in any area and affect patients regardless of their age, sex or ethnic origin. That is why they can be encountered by any doctor, both a specialist and a GP. Orphan Europe Group, , which deals with the development, registration, marketing and distribution of drugs applied in treating rare diseases delivers these products to the market. The so-called orphan drugs are medicinal products designated for treating rare diseases. The official European definition of a rare disease specifies it as a disease suffered by fewer than five in ten thousand people, a disease which is terminal or severely harmful to organs. At present there are over six thousand rare diseases known, which means that they affect millions of people –over 25 million patients in Europe only. So far drugs have been discovered for around 200-300 of these six thousand diseases. They are often genetic diseases, which means that they affect infants, children and young people. A doctor may never meet a patient with a rare disease. Therefore, there is always the concern that when a baby is born affected by this kind of disease, it will not be diagnosed properly and, in consequence, will not be properly treated.

Orphan Europe has been developing and marketing drugs used in rare disease treatment since 1990.

Orphan official website:

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