They are many rare genetic diseases that strike perhaps only one in a million people. Often incurable, they can be profoundly debilitating and frequently life-threatening. Though each particular disease is rare, they number in the thousands which means that together they affect about 30 million Europeans or around 7% of us. Treating these diseases is challenging and until recently, no cure was thought to be even possible for most of them. Now, hopes are rising for people living with rare genetic diseases as new treatments are being developed.

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