Multiple system atrophy is a rare, orphan status neurodegenerative disorder. It progresses rapidly leading to death of the affected individual within, on average, 6-9 years. There is currently no cure for the disease. Deposition of pathological α-Syn within oligodendroglial cells is its pathological hallmark. Therefore multiple system atrophy has been grouped with Parkinson´s disease into the family of synucleopathies. This provides a rationale for therapeutic and diagnostic approaches, which might result in progress in the clinical management of this rare disease.
Unlike Parkinson´s disease, where symptomatic treatments are well established, there are no drugs approved for the treatment of multiple system atrophy.
In multiple system atrophy, α-Syn builds up in the cytoplasm (known as glial cytoplasmic inclusions) of non-neuronal cells in the brain, called oligodendroglia, which provide support and protection to the nerve cells. As the disease progresses, the affected cells die off eventually leading to the onset of symptoms.