PONTOCEREBELLAR HYPOPLASIA TYPE 6 (PCH6)

Clinical trials for PONTOCEREBELLAR HYPOPLASIA TYPE 6 (PCH6) explained in plain language.

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Terminated 1

Experimental drug trial aims to tame devastating seizures in rare genetic disorders

Disease control Terminated

This study tested whether vatiquinone could reduce seizure frequency in people with mitochondrial diseases who have epilepsy that doesn't respond well to standard medications. It involved 68 participants who were randomly assigned to receive either the drug or a placebo for 24 we…

Matched conditions: PONTOCEREBELLAR HYPOPLASIA TYPE 6 (PCH6)

Phase: PHASE2, PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control

Last updated Apr 03, 2026 14:43 UTC

PONTOCEREBELLAR HYPOPLASIA TYPE 6 (PCH6)

Get alerted when new PONTOCEREBELLAR HYPOPLASIA TYPE 6 (PCH6) trials appear

Experimental drug trial aims to tame devastating seizures in rare genetic disorders