BioMarin Pharmaceutical commemorates 5 years of helping children with fatal rare disease
CLN2 Batten disease is a rare, inherited, fatal disorder that primarily affects the nervous system as a result of a failure to produce enough of the enzyme TPP1. The signs and symptoms of this condition typically start between the ages of 2 and 4, featuring recurrent seizures and coordination difficulty.
BioMarin Pharmaceutical in San Rafael developed a drug called Brineura five years ago that reduces the rate of decline in children afflicted with this batten disease, which is fatal, hereditary and comes in 13 types. The disorder impacts the body’s ability to get rid of cellular waste. The buildup on the cells causes the symptoms such as seizures, vision loss and problems with movement.