Cerliponase alfa, marketed as Brineura, is an enzyme replacement treatment for Batten disease, a neurodegenerative lysosomal storage disease. Specifically, Cerliponase alfa is meant to slow loss of motor function in symptomatic children over three years old with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). The disease is also known as tripeptidyl peptidase-1 (TPP1) deficiency, a soluble lysosomal enzyme deficiency. Approved by the United States Food and Drug Administration (FDA) on 27 April 2017, this is the first treatment for a neuronal ceroid lipofuscinosis of its kind, acting to slow disease progression rather than palliatively treat symptoms by giving patients the TPP1 enzyme they are lacking.The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. History
TPP1 was identified as the enzyme deficient in CLN2 Batten disease in 1997, via biochemical analysis that identified proteins missing a mannose-6-phosphate lysosomal targeting sequence. A gel electrophoresis was run for known brain proteins with lysosomal targeting sequences to see if a band was missing, indicating a deficiency in that protein. A band appeared to be missing at approximately 46 kDa, confirming its role in CLN2 disease, and almost the entire gene for this unknown protein was sequenced. The gene is located on chromosome 11. Today, it is known that varying mutation types occur in various locations of the gene including the proenzyme region, the mature enzyme region, or the signal sequence regions. After discovery, the recombinant form of TPP1, cerliponase alfa, was first produced in 2000, followed by testing in animal models until 2014.
A common symptom of this type is a high, spiking fever of 103 °F (39.4 °C) or higher, lasting for weeks or months, and a rash of pale red spots on the chest, thighs or other parts of the body may be visible. Cause
In most cases, juvenile arthritis is caused by the body attacking its own healthy cells and tissues, i.e. autoimmunity, causing the joint to become inflamed and stiff. Once the joint has become inflamed and stiff, damage is done to the joint and the growth of the joint may by changed or impaired. The underlying cause in the malfuction of the autoimmune system is unknown; dietary habits and emotional state seem to have no effect on the disease. Diagnosis
Early diagnosis and treatment by a paediatric rheumatologist or a rheumatologist can help manage inflammation, relieve pain, and prevent joint damage. However, it is difficult for doctors to diagnose the disease. Careful examination, laboratory tests (blood and urine), and various forms of imaging like X-rays may be some of the tests conducted by a doctor. Doctors may perform some of the following tests to diagnose the condition
ANA (Antinuclear Antibody) Test
Joint Aspiration
Rheumatoid Factor (RF) Test

Treatment
The treatment of juvenile arthritis includes medications, physical therapy, splints and in severe cases surgery. Methotrexate is commonly prescribed to children with juvenile arthritis. These treatments are focused on reducing swelling, relieving pain and maintaining full movement of joints. Children are encouraged to be involved in extra-curricular activities, physical activity when possible, and to live a "normal" life.