These high risk factors, i.e., young age, metastatic spread at diagnosis, presence of MYC amplifications, and prevalent LCA histology, all contribute to these tumors having the worst prognosis of any subgroup with an overall 5-year survival of <50%, especially in MYC-amplified tumors (Figure 2) [3,41,93,94]. The gene discussed is MYC; the disease is Leber congenital amaurosis.