Pharmaceutical composition comprising gamma-butyrobetaine for the treatment of syndromes induced by L-carnitine deficiency
AssigneeSigma-Tau Industrie Farmaceutiche Riunite S.p.A.
InventorClaudio Cavazza
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Abstract
Further to the discovery that the syndromes induced by L-carnitine deficiency are provoked by defects in the gamma butyrobetaine biosynthesis in skeletal and heart muscles, whereas the liver and kidney of the patients exhibiting such syndromes retain unaltered their L-carnitine-making ability, there are disclosed a gamma-butyrobetaine-containing composition and a therapeutical method based on gamma-butyrobetaine administration for the treatment of such patients.
Claims5
An orally or parenterally administrable pharmaceutical composition for the treatment of patients exhibiting L-carnitine deficiency syndromes, which comprises a therapeutically effective amount of gamma-butyrobetaine and a pharmacologically acceptable excipient.
A therapeutical method for the treatment of patients exhibiting L-carnitine deficiency syndromes, which comprises orally or parenterally administering to said patients a therapeutically effective amount of gamma-butyrobetaine.
The therapeutical method of claim 4, wherein said therapeutically effective amount of gamma-butyrobetaine is sufficient to biosynthetically produce in said patients an L-carnitine amount corresponding to normal plasma and tissue levels.
The therapeutical method of claim 5, which comprises orally or parenterally administering to said patients a gamma-butyrobetaine amount comprised between about 2 and about 20 mg/Kg of body weight/day.
Dependencies are inferred from the claim text.