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Jansky-Bielschowsky Disease
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Jansky-Bielschowsky Disease trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Jansky-Bielschowsky Disease trials may be worth asking aboutNorth America
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This is a first-in-human, open-label, single ascending dose study of TTX-381 for the treatment of ocular manifestations of CLN2 (Batten disease).
This study aims to assess the natural history of Batten disease (Neuronal Ceroid Lipofuscinosis) by obtaining information about the motor, behavioral, and funct…
This is an observational study that aims at assessing the natural history of NCL diseases as part of the international DEM-CHILD Database. 1. Patient data are…
The goal of this clinical trial is to learn if a gene therapy called scAAV9.CB.CLN6 can treat children with CLN6 Batten disease (variant late infantile neuronal…
This is a phase I/II randomized, masked, clinical trial to determine the safety and efficacy of intravitreal administration of cerliponase alfa.