Publication number: 20210252173
Abstract: Methods are provided for treating a subject having, or at risk of, a lysosomal storage disease, particularly Tay-Sachs, Gaucher disease, Sandhoff disease or Niemann-Pick disease, neuronal ceroid lipofuscinosis, or a condition associated with impaired Phospholipase A2 Group VI (PLA2G6) activity, particularly infantile neuroaxonal dystrophy or PLA2G6 associated neurodegeneration (PLAN), or a sleeping disorder, using a substituted polyunsaturated fatty acid, polyunsaturated fatty acid ester, polyunsaturated fatty acid thioester, fatty acid amide, polyunsaturated fatty acid mimetic, polyunsaturated fatty acid pro-drug, or combinations thereof, where the substituted compound comprises at least one substitution that reduces oxidation of the compound. Preferably, the substituted compound is a deuterated polyunsaturated fatty acid, or an ethyl ester thereof, such as 11,11-D2-linoleic acid, 11,11-D2-linoleic acid ethyl ester, 11,11,14,14-D4-linolenic acid, or 11,11,14,14-D4-linolenic acid ethyl ester.
Type:
Application
Filed:
April 18, 2019
Publication date:
August 19, 2021
Applicant:
Retrotope, Inc.
Inventors:
Mikhail S. SHCHEPINOV, Robert J. MOLINARI, Peter MILNER