Indication
Friedreich's ataxia
Friedreich's ataxia is a rare, inherited, progressive neurodegenerative disorder typically manifesting in late childhood or adolescence, though late-onset presentations can occur. Read more about Friedreich's ataxia
2 drugs 2 manufacturers
Drugs that treat it
Common Friedreich's ataxia therapies.
SKYCLARYS (omaveloxolone)
News & literature
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Coming soonFriedreich's ataxia is a rare, inherited, progressive neurodegenerative disorder typically manifesting in late childhood or adolescence, though late-onset presentations can occur.
It is inherited in an autosomal recessive pattern, most commonly caused by an expanded GAA trinucleotide repeat in the *FXN* gene, which results in a deficiency of the mitochondrial protein frataxin, impaired iron homeostasis, and mitochondrial energy dysfunction.
The disease predominantly affects the central and peripheral nervous systems—specifically causing degeneration of the dorsal root ganglia, sensory peripheral nerves, spinal cord tracts, and cerebellum—as well as non-neurological tissues.
Hallmark clinical features include progressive gait and limb ataxia, loss of deep tendon reflexes, dysarthria, sensory neuropathy, hypertrophic cardiomyopathy, scoliosis, and an increased risk of diabetes mellitus.
- medlineplus.gov
Friedreich's ataxia is a rare, inherited, progressive neurodegenerative disorder typically manifesting in late childhood or adolescence, though late-onset presentations can occur It is inherited in an autosomal recessive pattern, most commonly caused by an expanded GAA trinucleotide repeat in the *FXN* gene, which results in a deficiency of the mitochondrial protein frataxin, impaired iron homeostasis, and mitochondrial energy dysfunction
- rarediseases.org
Friedreich's ataxia is a rare, inherited, progressive neurodegenerative disorder typically manifesting in late childhood or adolescence, though late-onset presentations can occur The disease predominantly affects the central and peripheral nervous systems—specifically causing degeneration of the dorsal root ganglia, sensory peripheral nerves, spinal cord tracts, and cerebellum—as well as non-neurological tissues Hallmark clinical features include progressive gait and limb ataxia, loss of deep te...
- nih.gov
It is inherited in an autosomal recessive pattern, most commonly caused by an expanded GAA trinucleotide repeat in the *FXN* gene, which results in a deficiency of the mitochondrial protein frataxin, impaired iron homeostasis, and mitochondrial energy dysfunction The disease predominantly affects the central and peripheral nervous systems—specifically causing degeneration of the dorsal root ganglia, sensory peripheral nerves, spinal cord tracts, and cerebellum—as well as non-neurological tissues...
- nih.gov
It is inherited in an autosomal recessive pattern, most commonly caused by an expanded GAA trinucleotide repeat in the *FXN* gene, which results in a deficiency of the mitochondrial protein frataxin, impaired iron homeostasis, and mitochondrial energy dysfunction
- nih.gov
The disease predominantly affects the central and peripheral nervous systems—specifically causing degeneration of the dorsal root ganglia, sensory peripheral nerves, spinal cord tracts, and cerebellum—as well as non-neurological tissues
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