JOT109 project is designed to utilize JOTROL's advantages to treat patients with Mucopolysaccharidosis Type III, also known as SanFilippo Syndrome and "Children's Alzheimer's"
There is no treatment available for the disease today
JOTROL stimulates mitochondrial biogenesis, has CNS and systematic anti-inflammatory properties, improves neuronal function, and scavenges reactive oxygen species
These attributes should all have a positive effect in both MPSIII and Alzheimer’s patients
Target is to develop a treatment that will slow down and hopefully stop progression of the disease and thereby improve quality of life for the patient and their families
WHAT IS MPSIII, SANFILIPPO SYNDROME? IMAGINE ALZHEIMER’S, BUT IN CHILDREN.
In a nutshell, that’s what every family of children with Sanfilippo Syndrome faces. Our children are born with a change in their DNA that causes a very important enzyme to be made improperly … or not at all. Because they don’t have enough of this critical enzyme, our children cannot breakdown and recycle natural cellular waste. Their cells become clogged with toxic levels of heparan sulfate. While every cell in the body is affected, brain cells suffer the most. Effects on the brain become apparent between 2-6 years of age – speech problems, developmental delays, challenging behaviors, extreme hyperactivity, and poor sleep. Many children are initially diagnosed with Autism before finding the underlying diagnosis. Children will go on to experience progressive dementia similar to Alzheimer’s, stealing away their skills and knowledge. Children will often suffer from seizures. They will lose the ability to eat for themselves and many will get a feeding tube. They will lose the ability to walk and need a wheelchair. Children often pass away in their teenage years. It is relentless and devastating.
