Rare Diseases

Facts

6%-10% of the world’s population will suffer from a rare disease at one point in their life. Many of which have serious neurological manifestations. Early diagnosis of rare disorders is the key to halting disease progression. However, diagnosis of rare diseases can be challenging and is often delayed as a result of limited knowledge about these conditions among clinicians, as well as difficulties in accessing specialised services.

Finding a cure or improved treatments for such patients is fundamental not only to their lives but also to society as a whole. Patients with rare diseases feel excluded from the benefits of medical progress and argue forcefully that, because of the unmet medical need caused by rare diseases, these disorders deserve attention in their own right to address medical and pharmaceutical needs:

(1) the right to receive the best levels of care in the world;
(2) innovative therapies to patients suffering from rare diseases worldwide.