Infantile neuroaxonal dystrophy (INAD) is a rare pervasive developmental disorder that primarily affects the nervous system.

Phospholipid metabolism is important for many body processes, including helping to keep the cell membrane intact and functioning properly.

This impairment of enzyme function may disrupt cell membrane maintenance and contribute to the development of spheroid bodies in the nerve axons.

[citation needed] Mutations in the NAGA gene, resulting in alpha-N-acetylgalactosaminidase deficiency, cause an infantile neuroaxonal dystrophy known as Schindler disease.

Children with this disorder experience progressive deterioration of cognitive functions (dementia), and eventually lose awareness of their surroundings.

In some individuals with infantile neuroaxonal dystrophy, abnormal amounts of iron accumulate in a specific region of the brain called the basal ganglia.

[2] An open-label clinical study for long-term evaluation of efficacy, safety, tolerability, and pharmacokinetics of a deuterium-enhanced polyunsaturated fatty acid RT001, which, when taken with food, can protect the neuronal cells from degeneration, started in the Summer 2018.