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Most infants with Canavan disease appear normal for the first month of life

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How does gene therapy treat Canavan disease?

The goal of gene therapy is to restore normal amounts of aspartoacylase in the brain and nervous system and prevent the build-up of NAA and the symptoms of Canavan disease


What is the age group canavan disease affect?

Signs of Canavan disease normally become apparent around 6 months of age when an infant is not meeting normal milestones such as holding up his/her head. The lifespan for children with Canavan disease is generally 3-10 years if untreated, although some children have lived into their twenties.


What leads to Canavan disease?

It results when the body produces less than normal amounts of a protein called aspartoacylase


What is canavan disease?

Canavan deteriorates the white matter (myelin) in the brain. White matter is responsible for forming a protective coating around every nerve in the brain and spinal cord to ensure that nerve impulses are properly transmitted from one part of the body to another. Without myelin, the body cannot perform normal motor skills, can become increasingly stiff or floppy and suffers a multitude of symptoms including issues with swallowing, severe scoliosis and hip dislocations. Canavan Disease is a genetic disease, which means both parents must be carriers of the gene in order to have an affected child. If both parents are carriers, there is a 25% chance of a child having Canavan, a 50% chance of the child being a carrier, and a 25% chance of the child not having the Canavan gene at all.


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