Inborn disorder of amino acid transport
MONDO:0019216Also known as: inborn disorder of amino acid absorption and transport, disorder of amino acid absorption and transport
16 clinical trials for this condition and its sub-types.
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Broader categories
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New hope for kids with hard-to-treat genetic seizures
Disease control OngoingThis study tests a new medicine called PRAX-562 in 77 children with rare genetic epilepsies (SCN2A or SCN8A). The goal is to see if it safely reduces motor seizures. The trial has two parts: a double-blind phase where some children get the drug and some get a placebo, followed by…
Phase 2/3 • Sponsor: Praxis Precision Medicines • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Cystinuria drug trial pulled before it even started
Disease control CancelledThis study was designed to test whether ADV7103, a combination of potassium citrate and potassium bicarbonate, could help people with cystinuria by making their urine less acidic. The goal was to see if it could raise urine pH to 7.0 or higher, which might reduce kidney stone for…
Phase 2/3 • Sponsor: Advicenne Pharma • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC