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Friedreich's Ataxia
No estimate possible yet
Clinical trials
Omaveloxolone
Neurological disorders
New medicine (specialité)
Muscular diseases other
Reata
Oral
Capsule
Extramural (GVS)
Nrf2 activator
Centralised (EMA)
Normal trajectory
No
2023
2024
Yes
Revalidatie
Er is op dit moment nog geen directe behandeling mogelijk. De fase 2 studie is gepubliceerd in 2021 (1). Omaveloxolone verbeterde significant de neurologische functies vergeleken met placebo en was veilig.
continuous
1 times a day
2,5-300 mg
NCT02255435; Ann Neurol. 2021 Feb;89(2):212-225 (1);
< 340
Market share is generally not included unless otherwise stated.
Spierziekten.nl (1)
Friedreich's ataxia komt voor bij 1 op de 50.000 mensen (1). Voor Nederland zou dit dan gaan om 340 mogelijke patiënten.
There is currently nothing known about the expected cost.
There is currently nothing known about the possible total cost.
There is currently nothing known about off label use.
Mitochondrial disorders; Ocular inflammation; Ocular pain; Malignant melanoma
Adis insight
Huidige studies bevinden zich in fase 2.
There is currently no futher information available.
Understanding of expected market entry of innovative medicines