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Infantile Neuronal Ceroid Lipofuscinosis Clinical Trials

A listing of Infantile Neuronal Ceroid Lipofuscinosis medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.


Found (1) clinical trials

A clinical trial seeking patients for a research study for the treatment of Tay-Sachs Disease, Metachromatic leukodystrophy, Hunter's Syndrome, Niemann-Pick Disease, Sandhoff Disease, Neuronal ceroid lipofuscinosis, Adrenoleukodystrophy, Krabbe's Disease, alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses, Brain Diseases, Metabolic, Inborn, Pelizaeus-Merzbacher Disease

The inherited metabolic disorders (IMD) are a heterogeneous group of genetic diseases, most of which involve a single gene mutation resulting in an enzyme defect. In the majority of cases, the enzyme defect leads to the accumulation of substrates that are toxic and/or interfere with normal cellular function. Often times, ...