Neurogenetic Diseases

Read about Neurogenetic Diseases

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Description of Disease

Neurogenetic disease is caused by a defect in one or more genes which affects the nervous system.

Neurogenetic diseases include Huntington’s disease, Kennedy’s disease, spinocerebellar ataxia, spinal muscular atrophy, hereditary motor neuron disease, early onset muscle disorders, Charcot-Marie-Tooth neuropathies, hereditary spastic paraplegias, Friedreich’s ataxia, muscular dystrophies (Duchenne and LGMD) and congenital muscular dystrophies and myopathies.

Neurogenetic disease often results in a severe and often progressive disability. To diagnose neurogenetic disease, doctors typically review patients’ medical records, study their family history, interview and examine them, and conduct or obtain additional testing, such as genetic testing and neuro-imaging.

Although there are treatments to help manage some of these diseases, there are no cures. Gene therapeutic approaches provide promise for a cure for some of these diseases (NCBI).

Mysteries of Disease that Need to be Solved

Can we develop gene therapy to treat neuro-genetic disorders before they cause death and disability?

Support Research in Neurogenetic Diseases