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Background and Importance CLN2 disease (Neuronal Ceroid Lipofuscinosis Type 2) is an ultra-rare, neurodegenerative lysosomal storage disease, caused by an enzyme deficiency of tripeptidyl peptidase 1 (TPP1). Lack of disease awareness and the non-specificity of presenting symptoms often leads to delayed diagnosis. It has a devastating impact on children and families, leading to rapid functional decline and early death without effective treatment: early diagnosis and treatment of CLN2 disease are therefore vital to preserve function and slow decline for as long as possible. Cerliponase alfa, a recombinant form of the human TPP1 enzyme, is designed to replace the missing or deficient TPP1 enzyme in patients with CLN2 disease.Aim and Objectives This report will discuss the case of a patient who is responding to treatment, initiated in 2021.Material and Methods The patient is a little woman born in 2005. It was created of a custom pharmacological protocol, with continuous monitoring parameters vital, before during and after drug administration.Results The drug is stored at -20°C. Cerliponase alfa is administered, by clinician, by means of intracerebroventricular (ICV) infusion. The standard regimen involves a dose of 300 mg infused over approximately 4 hours, every 2 weeks. After verifying the clinical conditions, the doctor proceeds to prescribe the drug on the information system, on the date scheduled for administration. The treatment is taken care of and prepared at the Clinical Pharmacy laboratory. Once the chemical characteristics of the finished product have been verified, it is delivered to the department for subsequent administration.Conclusion and Relevance The Clinical Pharmacy Laboratory guarantees sterility and asepticity in the preparation of the drug. Administration is bimonthly, but temporary suspensions due to infections have occurred: the ICV delivery system, in fact, involves potential risks, in particular device-related complications, such as infections, which may require antibiotic treatment or device replacement. Despite this, the patient, in accordance with registered studies, appears to show a slower worsening of symptoms. This confirms the importance of encouraging and supporting clinical research, especially in rare diseases, which are often difficult to recognise and treat.Conflict of Interest No conflict of interest