Friedreichs-Ataxia-Information-Page

Friedreich Ataxia Information Page


What research is being done?

NINDS-funded researchers are studying the metabolic functions of mitochondria in individuals with Friedreich ataxia. Ongoing research is aimed at understanding the molecular basis for and mechanisms involved in the inactivation of the gene that provides instructions for frataxin and to develop signs that can indicate the diagnosis or progression of the disease. Researchers are investigating ways to turn off, or "silence," the defective FXN gene. Other research efforts include developing animal models of the disease that closely mimic the gene mutation and developing a protein replacement therapy for the disorder.

Information from the National Library of Medicine’s MedlinePlus
Friedrich's Ataxia

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What research is being done?

NINDS-funded researchers are studying the metabolic functions of mitochondria in individuals with Friedreich ataxia. Ongoing research is aimed at understanding the molecular basis for and mechanisms involved in the inactivation of the gene that provides instructions for frataxin and to develop signs that can indicate the diagnosis or progression of the disease. Researchers are investigating ways to turn off, or "silence," the defective FXN gene. Other research efforts include developing animal models of the disease that closely mimic the gene mutation and developing a protein replacement therapy for the disorder.

Information from the National Library of Medicine’s MedlinePlus
Friedrich's Ataxia

NINDS-funded researchers are studying the metabolic functions of mitochondria in individuals with Friedreich ataxia. Ongoing research is aimed at understanding the molecular basis for and mechanisms involved in the inactivation of the gene that provides instructions for frataxin and to develop signs that can indicate the diagnosis or progression of the disease. Researchers are investigating ways to turn off, or "silence," the defective FXN gene. Other research efforts include developing animal models of the disease that closely mimic the gene mutation and developing a protein replacement therapy for the disorder.

Information from the National Library of Medicine’s MedlinePlus
Friedrich's Ataxia


Definition
Definition
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