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Jansky-Bielschowsky Disease
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Jansky-Bielschowsky Disease trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Jansky-Bielschowsky Disease trials you may qualify forThis 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…
This is a phase I/II randomized, masked, clinical trial to determine the safety and efficacy of intravitreal administration of cerliponase alfa.
The purpose of this study is to gather information on the possible symptoms that patients with atypical neuronal ceroid lipofuscinosis type 2 (also known as aTP…
This is a multicenter, observational study for patients with a confirmed diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 d…