Methods of determining the presence and/or amount of a biomarker to determine the presence, identity, and/or severity of a lysosomal storage or neurological disorder
AssigneeBiomarin Pharmaceutical Inc.
InventorsBrett E. Crawford, Jillian R. Brown, Charles A. Glass, Jim R. Beitel, Robin M. Jackman
Life of this patent
Estimating the term…
Abstract
Provided herein are methods of diagnosing or monitoring the treatment of abnormal glycan accumulation or a disorder associated with abnormal glycan accumulation.
Claims21
A method of determining the presence of and/or measuring the amount of a biomarker to determine the presence, identity, and/or severity of the disease or condition in an individual having a disease or condition associated with abnormal glycan biosynthesis, degradation, or accumulation, the method comprising:
- (a) generating the biomarker comprising of one or more non-reducing end glycan residual compound(s), wherein the biomarker is generated by treating a population of glycans, in or isolated from a biological sample from the individual, with at least one digesting glycan enzyme(s), wherein prior to enzyme treatment, the biomarker is not present in abundance in samples from individuals with the disease or condition relative to individuals without the disease or condition, and
- (b) using an analytical instrument to detect the presence of and/or measure the amount of the biomarker produced and displaying or recording the presence of or a measure of a population of the biomarker;
- and
- (c) determining the presence, identity, and/or severity of the disease or condition utilizing the presence of and/or measure the amount of the biomarker;
- wherein the disease or condition is a lysosomal storage disorder or a neurological disorder;
- and
- wherein when the lysosomal storage disorder is an MPS disorder, then the digesting glycan enzyme is not a lyase.
A method monitoring the treatment of a disorder associated with the abnormal degradation, biosynthesis and/or accumulation of glycans, the method comprising:
- (a) following administration of an agent for treating a disorder associated with the abnormal degradation, biosynthesis and/or accumulation of glycans to an individual in need thereof, using an analytical instrument to measure the amount of a population of a biomarker comprising non-reducing end glycan residual compounds present in a transformed biological sample, the biomarker being generated by treating a population of glycans, in or isolated from a biological sample from the individual, with at least one digesting glycan enzyme(s), wherein prior to enzyme treatment, the biomarker is not present in abundance in samples from individuals with the disease or condition relative to individuals without the disease or condition, and
- (b) determining whether or not the amount of biomarker has decreased or increased at a slower rate compared to the amount or rate of increase prior to administration of the agent for treating a disorder associated with the abnormal degradation, biosynthesis and/or accumulation of glycans;
- and
- wherein the disorder is a lysosomal disorder or a neurological disorder.
Dependencies are inferred from the claim text.