BATTEN DISEASE
Clinical trials for BATTEN DISEASE explained in plain language.
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New cell booster may help cord blood transplants reach the brain in kids with rare diseases
Disease control Recruiting nowThis early-stage study tests whether adding special cells (DUOC-01) to a standard cord blood transplant is safe for children with inherited metabolic diseases that damage the brain. About 40 participants, from 1 week to 21 years old, will receive the cells via a spinal injection.…
Matched conditions: BATTEN DISEASE
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated May 26, 2026 12:04 UTC
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New study aims to unlock secrets of rare, deadly batten disease
Knowledge-focused Recruiting nowThis study is for people with Batten disease (CLN3) and their family members. Researchers want to collect samples like blood, spinal fluid, and skin, and track symptoms over time. The goal is to find biological markers that can be used to measure the effects of future treatments.…
Matched conditions: BATTEN DISEASE
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated May 26, 2026 12:01 UTC
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Scientists watch rare brain diseases unfold in 1,500 patients
Knowledge-focused Recruiting nowThis study follows 1,500 people with rare genetic brain disorders like MLD, Krabbe disease, and Batten disease. Researchers track thinking, movement, and language skills over time, and collect blood and MRI scans. The goal is to learn how these diseases progress, not to test a tr…
Matched conditions: BATTEN DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated May 26, 2026 10:35 UTC
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Join the fight against blindness – share your story
Knowledge-focused Recruiting nowThis registry collects health information from people with inherited retinal diseases to help researchers better understand these conditions. Participants create an online profile and can update it over time. The goal is to speed up research and connect people with clinical trial…
Matched conditions: BATTEN DISEASE
Sponsor: Foundation Fighting Blindness • Aim: Knowledge-focused
Last updated May 22, 2026 14:04 UTC
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Batten disease study seeks 500 participants to track disease progression
Knowledge-focused Recruiting nowThis study follows children and adults with Batten disease to learn how the condition changes over time. Researchers will track movement, behavior, and daily skills using special tests. The goal is to better understand the disease and improve how it is measured, which may help fu…
Matched conditions: BATTEN DISEASE
Sponsor: University of Rochester • Aim: Knowledge-focused
Last updated May 19, 2026 12:00 UTC