Sideroblastic anemia 3
MONDO:0014804Also known as: GLRX5-related sideroblastic anaemia, GLRX5-related sideroblastic anemia, SIDBA3, adult-onset autosomal recessive sideroblastic anaemia, adult-onset autosomal recessive sideroblastic anemia, anemia, sideroblastic, 3, pyridoxine-refractory
80 clinical trials for this condition and its sub-types.
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New drug aims to tame hard-to-control seizures in rare mitochondrial disorders
Disease control TerminatedThis study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …
Phase: PHASE2, PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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Can extra care after transplant help older patients live better?
Disease control TerminatedThis study tested two extra care programs—supportive palliative care and clinical management of other health issues—for older or frail patients who had a donor stem cell transplant. The goal was to see if these programs could improve quality of life and survival compared to stand…
Phase: PHASE2, PHASE3 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC