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Neuroaxonal Dystrophy, Atypical
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Neuroaxonal Dystrophy, Atypical trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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Mutations in the PLA2G6 gene are well known in the classical phenotype called infantile neuro-axonal dystrophy (INAD), a severe neurodegenerative disease starti…
This study is a longitudinal and prospective study of the natural history of infantile neuroaxonal dystrophy (INAD).
The purpose of this study is to evaluate the efficacy and safety of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD).