Lack of Information on the Effects of COVID-19 on Rare Pathologies Has Further Hampered Access to Healthcare Services

Classic and Atypical Late Infantile Neuronal Ceroid Lipofuscinosis in Latin America: Clinical and Genetic Aspects, and Treatment Outcome with Cerliponase Alfa

“…Discontinuation due to issues related to access to the medication underscores the importance of ensuring that these patients are treated consistently and of avoiding the effects of access inequalities or healthcare barriers, as discontinuation might reduce treatment effectiveness and, consequently, patient functionality. In 5.5% of cases, discontinuation was attributed to the SARS-Cov2 pandemic which likely disrupted drug administration, as reported for rare diseases [ 40 ]; 13.9% of patients discontinued the medication due to infection, and all of them reinitiated later, with no other adverse events leading to discontinuation or drug-related events being reported.…”