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Machado-Joseph disease
MONDO:0007182Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease, is the most common subtype of type 1 autosomal dominant cerebellar ataxia (ADCA type 1), a neurodegenerative disorder, and is characterized by ataxia, external progressive ophthalmoplegia, and other neurological manifestations.
Also known as: Azorean disease of the nervous system, MJD, Machado disease, Machado-Joseph disease, Nigro-spino-dentatal degeneration with nuclear ophthalmoplegia, SCA3, autosomal dominant striatonigral degeneration, spinocerebellar ataxia 3
30 clinical trials for this condition and its sub-types, 11 tagged with Machado-Joseph disease itself.
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Browse by category →Sub-types of Machado-Joseph disease
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Machado-Joseph disease type 3 2 trials
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Machado-Joseph disease type 1 0 trials
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Machado-Joseph disease type 2 0 trials
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Machado-Joseph disease type 4 0 trials
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New drug could slow rare brain disease that steals balance
Disease control OngoingThis phase 3 trial tests whether the drug troriluzole can slow the progression of spinocerebellar ataxia, a rare genetic disorder that affects coordination and balance. About 300 adults with different types of SCA are randomly assigned to take either troriluzole or a placebo dail…
Phase 3 • Sponsor: Biohaven Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Could a drug slow rare brain disease? new study uses Real-World data to find out
Disease control OngoingThis study looks at whether the drug troriluzole can slow the progression of spinocerebellar ataxia (SCA), a rare genetic disease that affects movement and balance. Researchers will compare 909 patients who took troriluzole for up to three years with similar patients who did not …
Sponsor: Biohaven Therapeutics Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:04 UTC