Spastic ataxia 4
MONDO:0013354Any autosomal recessive spastic ataxia in which the cause of the disease is a mutation in the MTPAP gene.
Also known as: MTPAP autosomal recessive spastic ataxia, SPAX4, autosomal recessive spastic ataxia caused by mutation in MTPAP, autosomal recessive spastic ataxia type 4, spastic ataxia type 4, autosomal recessive spastic ataxia - optic atrophy - dysarthria, autosomal recessive spastic ataxia 4, autosomal recessive spastic ataxia-optic atrophy-dysarthria syndrome
14 clinical trials for this condition and its sub-types.
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New drug shows promise for rare energy disorder
Disease control CompletedThis study tested a new drug called OMT-28 in 28 people with primary mitochondrial disease, a condition that affects how cells produce energy. Participants took the drug once daily for 6 months, and researchers measured safety, blood markers of inflammation, and symptoms like fat…
Phase 2 • Sponsor: Omeicos Therapeutics GmbH • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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Long-Term safety of mitochondrial drug confirmed in 101 patients
Disease control CompletedThis study looked at the safety of vatiquinone in 101 people with inherited mitochondrial disease who had already taken the drug in a previous study or treatment plan. The goal was to track any side effects until the drug became commercially available or the program ended. Partic…
Phase 3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC