Neuronal ceroid-lipofuscinoses
Clinical trials for Neuronal ceroid-lipofuscinoses explained in plain language.
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Rare brain disease study seeks to unlock mysteries of atypical TPP1 deficiency
Knowledge-focused OngoingThis study follows 5 people with a rare, late-onset form of TPP1 deficiency (a brain disease) to track how their symptoms change over time. Researchers will use tests like brain scans, eye exams, and movement assessments to better understand the condition. The goal is to gather i…
Matched conditions: NEURONAL CEROID-LIPOFUSCINOSES
Sponsor: Children's Hospital of Orange County • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:05 UTC
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New study aims to measure how CLN2 disease affects Children's development
Knowledge-focused By invitation onlyThis study looks at how children with CLN2 Batten disease develop over time, including their thinking, language, and motor skills. Researchers will compare children who receive treatment (cerliponase alfa) with those who do not. The goal is to find better ways to measure the dise…
Matched conditions: NEURONAL CEROID-LIPOFUSCINOSES
Sponsor: Jessica Scherr • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC