Current Drugs Strategies for Treatment of Rare Neurodegenerative Diseases

Abstract

Nervous system problems affect around 600 million people worldwide. Among these, neurodegenerative illnesses are often distinguished by a late adult start, a progressive clinical course, and a localized loss of neurons in the central nervous system. These include, among others, multiple sclerosis, Parkinson’s disease, amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease), Huntington’s disease, Prion diseases, encephalitis, epilepsy, genetic brain disorders, hydrocephalus, stroke, and Alzheimer’s and other less common dementias. The brain stem, cerebellum, thalamus, hypothalamus, basal ganglia, cerebral cortex, and intracranial white matter are among the areas that neurodegeneration typically affects. Mendelian inheritance is well-established, despite the fact that most neurodegenerative illnesses are sporadic. The neuropathological findings and clinical symptoms in hereditary neurodegenerative disorders are intriguing. Regretfully, there are few neurodegenerative diseases for which no effective treatments are available. The rare hereditary types of neurodegenerative diseases, such as ataxias, multiple system atrophy, spastic paraplegias, Parkinson’s disease, dementias, motor neuron diseases, and uncommon metabolic disorders, are highlighted in this chapter along with their clinical and genetic characteristics.