Clinical Trials

4 trials
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Recruiting × Observational × Genetic Diseases, Inborn × Clear all
NCT04880356 2024-11-19

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

Recruiting
100 enrolled

2-Hydroxyglutaricaciduria, Fahr's disease, Polycystic lipomembranous osteodysplasia with sclerosing leukoencephalopathy, Methylmalonic acidemia with homocystinuria, Undiagnosed Diseases, Adrenoleukodystrophy, Cysts, Hepatolenticular Degeneration, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Metabolic Diseases, Nervous System Diseases, Sphingolipidoses, Xanthomatosis, Cerebrotendinous, Pelizaeus-Merzbacher Disease, Genetic Diseases, Inborn, Rare Diseases, Alexander Disease, Leukoencephalopathies