Methods of treating mitochondrial dysfunction

A dysfunctional, mitochondrial technique for use in the diagnosis and treatment of human subjects with mitochondrial dysfunction or mitochondrial disease, inherited mitochondrial disease, and computing said composite measurements in the field, capable of addressing issues that have not been proposed in multiple organs or a wide range of Spectrum therapeutics and other issues

Pending Publication Date: 2021-06-18
RIBONOVA INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

In this work, probucol was proposed to target only pancreatic beta cells, and thus was not proposed as a multi-organ or broad-spectrum therapeutic agent

Method used

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  • Methods of treating mitochondrial dysfunction
  • Methods of treating mitochondrial dysfunction
  • Methods of treating mitochondrial dysfunction

Examples

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example 1

[0118] Example 1: Phase II Randomized, Double-Blind, Placebo-Controlled, Two-Period Crossover Study to Evaluate the Safety and Efficacy of Probucol in Adults with Genetically Confirmed Mitochondrial Diseases

[0119] Non-limiting examples of clinical trials evaluating the efficacy of probucol, or a pharmaceutically acceptable salt thereof, in subjects with genetically confirmed mitochondrial disease are described below.

[0120] A. Research purpose

[0121] main purpose

[0122] Among other things, the primary objective of this study was to evaluate (i) the ability of subjects who were responders during the 16-week treatment period to be assessed by the Mitochondrial Disease Personalized Composite (MitoPC) endpoint compared to placebo Probucol or its pharmaceutically acceptable salt efficacy of ratio, described measurement is based on the most prevalent symptom in the patient suffering from mitochondrial disease, and (ii) use adverse event (AE) incidence rate, laboratory valu...

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Abstract

This disclosure is directed to methods of treating or preventing mitochondrial dysfunction or mitochondrial disease in a subject comprising administration of probucol, or a pharmaceutically acceptable salt thereof. This disclosure is also directed to methods of diagnosing genetic mitochondrial disease in a subject prior to and in association with said treatment or prevention. This disclosure is also directed to methods of assessing and managing a subject with mitochondrial dysfunction or mitochondrial disease using a composite measurement.

Description

technical field [0001] Disclosed herein are methods of treating or preventing mitochondrial dysfunction or mitochondrial disease in a subject, the methods comprising administering probucol or a pharmaceutically acceptable salt thereof. Also disclosed herein are methods of diagnosing an inherited mitochondrial disease in a subject prior to and in connection with said treatment or prophylaxis. Also disclosed herein are methods of managing a patient with mitochondrial dysfunction or mitochondrial disease and assessing the patient's response to a therapeutic intervention by using a composite measurement that combines results from two or more sub-instruments. Also disclosed herein is a method of calculating the composite measurement. Further disclosed herein is an integrated method for diagnosing and treating a human subject with mitochondrial dysfunction or a mitochondrial disease and assessing the patient's response using the composite measurement. Background technique [000...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K31/12A61K31/337A61K31/352
CPCA61P21/00A61K31/10A61K9/00A61K45/06C12Q1/6883A61B5/1124A61B5/4076C12Q2600/156
Inventor M·J·法尔克S·E·麦科马克Z·佐尔基普利-坎宁安N·L·韦布E·C·袁A·W·福德-哈钦森
Owner RIBONOVA INC
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