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Ceroid Lipofuscinosis, Neuronal 5
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Ceroid Lipofuscinosis, Neuronal 5 trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Ceroid Lipofuscinosis, Neuronal 5 trials you may qualify forThis study aims to assess the natural history of Batten disease (Neuronal Ceroid Lipofuscinosis) by obtaining information about the motor, behavioral, and funct…
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, i…
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…
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple…
This is a prospective, non-randomized, open-label, dose escalation study of a single administration of gene therapy in children who are 3 to 9 years old with Ne…
CLN5 is a form of Batten Disease, a neurodegenerative disorder in children causing psychomotor regression, seizures, blindness, loss of ambulation and premature…