Purpose: Cerebrotendinous xanthomatosis (CTX) is a rare genetic lipid storage disorder of bile acid synthesis characterized by a diverse range of clinical signs and symptoms that can manifest early in life. Affected individuals may experience infantile diarrhea and jaundice, childhood cataracts, tendon xanthomas, developmental delays, intellectual disabilities, and neurological symptoms. The complications can be severe, leading to significant motor dysfunction, cognitive impairment, and dementia. Timely diagnosis and initiation of bile acid treatment are essential for improving long-term outcomes and preventing serious neurological decline in patients with CTX. Treatment with chenodeoxycholic acid (CDCA) or cholic acid (CA) corrects abnormal bile acid synthesis, can alleviate clinical signs and symptoms, and prevent further deterioration. Larger cohort studies in older patients have demonstrated the importance of early diagnosis and prompt CDCA treatment for improving patient long-term outcomes. There is no data available from a larger cohort study assessing the effect of early treatment in infants or young children with CTX, although a number of case reports describing treatment in infants and children with CTX indicate that long-term bile acid treatment is safe and can prevent development of the signs and symptoms of CTX. Methodology: We reviewed existing literature on the outcomes of CTX-affected patients treated from infancy. Additionally, we included insights from our experiences in Israel treating eight CTX affected newborns with CDCA or CA from infancy (0–6 months) onward. Conclusion: Our real-world data indicate that treating CTX with bile acids in the early months of life is safe with careful clinical monitoring and that patients treated early in life experience better health outcomes compared to those treated later in childhood or adulthood. Striking and significant differences in growth parameters were also observed, with growth parameters significantly improved in childhood for patients treated from infancy onward. Based on this data, as well as a review of the latest literature, and in accordance with CTX expert consensus guidelines that recommend to initiate treatment from birth onward to prevent the accumulation of harmful metabolic intermediates, we advise initiating treatment within the first few months of life for patients identified in infancy through family history, genetic testing, or newborn screening for CTX. We believe that the evidence compiled here will enhance the timing of optimal treatment for this condition, particularly for affected infants who may be identified in the future. Furthermore, the implementation of universal newborn screening for CTX will be critical to facilitate early identification and intervention during infancy, leading to improved health outcomes for affected patients and a reduction in the morbidity and mortality associated with the untreated progression of this serious disease.