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US 2019/0161535 A1Published application
Compositions and methods for treating spinal muscular atrophy
AssigneeAnnexon, Inc.
InventorsTed Yednock, Sethu Sankaranarayanan
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Abstract
The present disclosure relates generally to methods of preventing, reducing risk of developing, or treating spinal muscular atrophy, comprising administering to a subject an inhibitor of the complement pathway.
Claims29
A method of preventing, reducing risk of developing, or treating spinal muscular atrophy (SMA), comprising administering to a subject an inhibitor of the complement pathway.
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A method of determining a subject's risk of developing spinal muscular atrophy, comprising:
- (a) administering an anti-C1q, anti-C1r, or anti-C is antibody to the subject, wherein the anti-C1q, anti-C1r, or anti-C1s antibody is coupled to a detectable label;
- (b) detecting the detectable label to measure the amount or location of C1q, C1r, or C1s in the subject;
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- (c) comparing the amount or location of one or more of C1q, C1 r, or C s to a reference, wherein the risk of developing spinal muscular atrophy is characterized based on the comparison of the amount or location of one or more of C1q, C1r, or C1s to the reference.
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Dependencies are inferred from the claim text.