BATTEN DISEASE
Clinical trials for BATTEN DISEASE explained in plain language.
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Gene therapy survivors tracked for years in rare brain disease study
Knowledge-focused OngoingThis study follows 10 people with CLN6 Batten disease who previously received a one-time gene therapy injection into the spine. Researchers are checking for long-term side effects and using a standard scale to measure how the disease progresses over time. The goal is to understan…
Matched conditions: BATTEN DISEASE
Sponsor: Emily de los Reyes • Aim: Knowledge-focused
Last updated May 17, 2026 04:37 UTC
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New study aims to measure how CLN2 affects Kids' skills over time
Knowledge-focused ENROLLING_BY_INVITATIONThis study follows 30 children with CLN2 Batten disease to measure changes in thinking, movement, and language skills over time. Researchers will compare children who receive the treatment cerliponase alfa with those who do not. The goal is to find better ways to track the diseas…
Matched conditions: BATTEN DISEASE
Sponsor: Jessica Scherr • Aim: Knowledge-focused
Last updated May 13, 2026 16:03 UTC