Single-Patient Therapies: Between Hope and Tragedy

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Just a year ago, a case was published of a baby, K.J. Muldon, who suffered from a rare genetic metabolic disease – which can cause irreversible neurodegeneration and be fatal – and was cured thanks to personalized gene therapy. A pioneering example of the so-called n=1 therapy, that is, a therapy specifically created to treat a specific mutation that only one patient has. This success story was considered one of the great scientific milestones o…