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94 results for “CLN3”

Trials

View all 59 →

Active, not recruiting

Gene Therapy for Children With CLN3 Batten Disease

7 participants · First posted Dec 10, 2018

Not yet recruiting

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

24 participants · First posted Jul 31, 2026

Investigations of Juvenile Neuronal Ceroid Lipofuscinosis

300 participants · First posted Oct 11, 2017

Drugs

View all 19 →

CLN7(Neurogene)

CLN-619

CLN-049

News

View all 16 →

New Retina Model Reveals How CLN3-Batten Disease Damages Vision and Points to a Treatment

Batten-1 Advances Toward Pivotal Phase 3 Trial as THX Pharma, Biocodex, and BBDF Unite on International Batten Disease Awareness Day

Azafaros to Present Phase 3 Nizubaglustat Data for Rare Lysosomal Storage Disorders at WORLDSymposium 2026

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