One patient gets experimental enzyme to fight rare kidney disease

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

* INCLUSION CRITERIA: Subject must meet all of the following inclusion criteria to be eligible for enrollment into the study: * Greater than or equal to 18 years at screening; * Confirmed FLD diagnosis through genotype confirmation of a LCAT mutation, as documented at screening or from previous testing; * History of HDL-C \< 15 mg/dL over the past year; * Serum creatinine \> 3 mg/dL; * Increasing renal dysfunction resulting in ESRD; * Chronic concomitant medications must be stable for at least 2 weeks prior to screening (for example, lipid-altering drugs and/or ACE-inhibitors used for the treatment of FLD). * Subject is willing and able to comply with scheduled study visits and is able to tolerate study procedures, including weekly to bi-weekly infusions over 1 year. * Subject must be able to provide a personally-signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study. * Subject may be currently on dialysis or require dialysis during the study EXCLUSION CRITERIA: Subject will not be included in the study if he presents with any of the following: * History of febrile illness within 5 days prior to dosing; * Active non-basal cell cutaneous malignancy requiring surgery, chemotherapy, and/or radiation in the past 12 months. NOTE: Subjects with localized prostate cancer under a watchful-waiting treatment plan without evidence of disease progression in the past year may participate in the study if approved by the investigator and sponsor or designee. NOTE: Subjects diagnosed with basal cell carcinoma of the skin within the past 12 months must receive adequate treatment for their basal cell skin carcinoma prior to randomization. * Current documented drug or alcohol abuse that would interfere with the subject s compliance with study procedures; * Treatment with an investigational drug within 28 days prior to dosing. * Known hypersensitivity to heparin or IV infusion equipment, plastics, adhesive or silicone or known history of hypotension or infusion site reactions with IV administration. * Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and, in the judgment of the investigator, would make the subject inappropriate for entry into this trial or compromise the subject s ability to continue with the procedures of the trial.