Friedreich’s ataxia (FRDA) is an inherited autosomal recessive genetic condition that progressively damages the nervous system, affecting the people between 10 and 15 years of age. The risk factor of Friedreich's ataxia is diabetes mellitus and heart diseases including cardiomyopathy and cardiac arrhythmias. Friedreich's ataxia occurs due to mutation in the frataxin (FXN) gene, which is inherited from both parents, leading to the presence of mutations in both copies of FXN gene. The initial symptoms of Friedreich’s ataxia include frequent falling, difficulty in walking owing to impairment in the coordination ability voluntary movements (ataxia) and unsteady posture. People with Friedreich's ataxia also develop characteristic foot deformities, unbalanced sideways or lateral curvature of the spine (scoliosis), degeneration in the sensory nerve fibers at dorsal root ganglia, impaired swallowing, and slurred speech (dysarthria).
Friedreich’s ataxia is diagnosed by molecular genetic testing to detect mutations in the FXN gene. The symptomatic treatment of Friedreich's ataxia includes continuous medical supervision to avoid further complications involving the spine, heart, muscles, feet, hearing and vision. Genetic counseling is also recommended for affected individuals and their families. Wheelchairs, prostheses, physical therapy and walking aids can also help to improve patient’s lifestyle. Non-surgical interventions or orthopedic surgery can help in the treatment of the feet and curvature of the spine abnormalities, with proper consultation with an orthopedic surgeon and neurologist. Heart problems associated with Friedreich's ataxia may be treated with anti-cardiac failure medication and anti-arrhythmic medication. Diabetes mellitus associated with Friedreich's ataxia can be treated with an oral hypoglycemic therapeutics, dietary modification and insulin.
Various companies are developing drug candidates using different technologies for the development of effective and better drugs, which is driving the growth of the pipeline.
As of July 2017, the Friedreich's ataxia therapeutics pipeline comprises of approximately 17 drug candidates in different stages of development.
FRIEDREICH'S ATAXIA DRUG CANDIDATES UNDER DEVELOPMENT (2017)
Some of the key players developing drugs for the treatment of Friedreich's ataxia include Cardero Therapeutics, Inc., Edison Pharmaceuticals, Inc., Retrotope, Inc. and others.
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