BATTEN DISEASE
Clinical trials for BATTEN DISEASE explained in plain language.
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New hope for kids with devastating brain diseases: experimental cell therapy aims to halt progression
Disease control Recruiting nowThis early-stage study is testing the safety of adding a special type of cell, derived from umbilical cord blood, to a standard cord blood transplant for children with rare inherited metabolic diseases that damage the brain. The goal is to see if delivering these cells directly i…
Matched conditions: BATTEN DISEASE
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC
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Scientists track rare childhood brain disease to find answers
Knowledge-focused Recruiting nowThis study aims to understand how Batten disease progresses over time by tracking 500 children and adults with the condition. Researchers will use regular check-ups and questionnaires to measure changes in movement, thinking, behavior, and daily function. The goal is to create be…
Matched conditions: BATTEN DISEASE
Sponsor: University of Rochester • Aim: Knowledge-focused
Last updated Apr 01, 2026 22:56 UTC
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Scientists gather clues in fight against rare, fatal childhood brain disease
Knowledge-focused Recruiting nowThis study aims to collect information and biological samples from people with Batten disease (CLN3) and their family members. The goal is to identify measurable signs, or 'markers,' of the disease that can be used to test future treatments in clinical trials. Researchers will fo…
Matched conditions: BATTEN DISEASE
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:32 UTC
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Scientists launch major effort to map progression of devastating rare brain diseases
Knowledge-focused Recruiting nowThis study aims to understand how over 25 rare genetic disorders that damage the brain progress over time. Researchers will follow 1,500 participants, assessing their development and brain health regularly for years. The goal is to gather detailed information to help future resea…
Matched conditions: BATTEN DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC