Use of nikkomycin for the preparation of a medicament for the treatment of a lipid storage myopathy
Nicotinycin addresses the treatment challenges of NLSDM by activating the autophagy pathway and upregulating ATGL protein expression, providing a safe and economical treatment option that significantly reduces lipid droplet deposition and inflammatory response, and improves energy production.
CN121891373BActive Publication Date: 2026-07-10SHANDONG UNIV QILU HOSPITAL
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Patent Information
- Application Number
- CN202610360811.5
- Authority / Receiving Office
- CN · China
- Patent Type
- Patents(China)
- Current Assignee / Owner
- Filing Date
- 2026-03-24
- Publication Date
- 2026-07-10
- Estimated Expiration
- 2046-03-24
AI Technical Summary
Technical Problem
Currently, there are no effective drugs for treating simple myopathy-type neutral fat storage disease (NLSDM), existing treatments are not effective, and enzyme replacement therapy is costly and carries unknown risks.
Method used
Using nicotinic acid as a therapeutic agent, lipid droplet deposition was significantly reversed, inflammatory response was reduced, autophagy inhibition was improved, and energy production was increased by activating the autophagy pathway and upregulating ATGL protein expression.
Benefits of technology
Nicotinamide significantly reduces lipid droplet deposition, improves energy production in NLSDM patients, reduces inflammatory response, and provides a safe and economical treatment option.
✦ Generated by Eureka AI based on patent content.
Abstract
This invention belongs to the pharmaceutical field and relates to the application of nikkomycin in the preparation of drugs for treating lipid storage myopathy. This invention discloses the use of nikkomycin in the treatment of lipid storage myopathy, particularly in the treatment of simple myopathic neutral lipopathy caused by PNPLA2 gene mutations. Simple myopathic neutral lipopathy is an autosomal recessive, progressive lipid storage myopathy affecting multiple systems throughout the body. Currently, there is no treatment available, and patients develop fatal symptoms such as generalized muscle weakness and cardiomyopathy in the late stages. This invention discovers that nikkomycin can significantly reverse lipid droplet deposition in fibroblasts of patients with simple myopathic neutral lipopathy, reduce inflammatory responses, improve autophagy inhibition, and increase energy production. It can serve as an effective ingredient for treating this disease and has clear application prospects in patients with untreatable simple myopathic neutral lipopathy.
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