Study for the Treatment for CLN7 Disease

Inclusion Criteria: * 1-18 years of age * Clinically symptomatic patients with diagnosis of CLN7 based upon molecular testing with homozygous or compound heterozygous and pathogenic mutations in MFSD8 gene with symptom onset before age 4 * Patients selected to be included in this study will have no more than moderate severity of the disease and will have to meet the following criteria; Not dependent on chronic invasive ventilatory support AND have either * Expressive language sub test on Mullen and/or Vineland consistent with an age equivalent score of a 2 year 0 month old. This means they should have 20-50 words (all comprehensible) in their vocabulary and putting 2-3 words phrases in a sentence or, * Patients can complete and obtain a score of 2-3 on GMFM sub domain E (Walking, Running \& jumping) item 67 (ie With 2 hands held can walk 10 steps forward) * Written informed consent provided by participant/parent/guardian and willingness to participate and comply with all the study related visits and procedures. Assent provided by children 10 -17 years old based on their ability to understand the risks and possible benefits, and the activities expected of them as participants. Exclusion Criteria: * Diagnosis of a second neurodegenerative disease or another genetic syndrome with a progressive course * Hypersensitivity to any drugs used per procedural protocol * Inability to tolerate anesthesia or study procedures * Advanced stage disease defined by the use of chronic invasiv…