Examples of patient-customized ASOs include Milasen, which was used to treat a 6-year-old girl with Batten’s disease (caused by a pathogenic mutation in CLN7) and Atipeksen, which was used to treat ataxia telangiectasia in a 3-year-old boy [168,253]. The gene discussed is MFSD8; the disease is juvenile neuronal ceroid lipofuscinosis.