NEURONAL CEROID LIPOFUSCINOSIS TYPE 2
Clinical trials for NEURONAL CEROID LIPOFUSCINOSIS TYPE 2 explained in plain language.
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Eye injection may save sight in kids with rare batten disease
Disease control OngoingThis study tests whether injecting a replacement enzyme directly into the eye can slow or stop vision loss in children with CLN2 Batten disease, a rare genetic disorder that causes blindness and brain damage. Five children aged 2 to 6 who already receive brain infusions of the en…
Matched conditions: NEURONAL CEROID LIPOFUSCINOSIS TYPE 2
Phase: PHASE1, PHASE2 • Sponsor: David L Rogers, MD • Aim: Disease control
Last updated Jun 27, 2026 12:26 UTC
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Rare disease drug safety tracked in new observational study
Knowledge-focused OngoingThis study follows 35 children with CLN2 disease (a rare, severe brain disorder) who are taking cerliponase alfa (Brineura). Researchers will monitor for side effects and serious reactions over time. The goal is to better understand the long-term safety of this enzyme replacement…
Matched conditions: NEURONAL CEROID LIPOFUSCINOSIS TYPE 2
Sponsor: BioMarin Pharmaceutical • Aim: Knowledge-focused
Last updated Jul 02, 2026 00:00 UTC