Phosphopantothenate has been shown to treat<unk>  in a human, and also in a mouse model of the disease. Pantethine (a precursor of pantetheine) has been studied and shown to be effective in a mouse and in a fruit fly model of the disease.
Pantothenate kinase-associated neurodegeneration
Cerebral creatine deficiency
Neuronal ceroid lipofuscinosis
N-Acetylglutamate synthase deficiency
Autoimmune GFAP astrocytopathy
Dopamine beta hydroxylase deficiency
Glycogen storage disease
Lysosomal storage disease