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Introduction

Drug repurposing, also known as drug repositioning or drug reprofiling, is the process of finding new uses for existing drugs outside the scope of their original medical indications. This approach has gained significant attention in recent years due to its potential to bring new treatment options to patients with rare genetic disorders, for which traditional drug development strategies have been largely unsuccessful. Rare genetic disorders, also known as orphan diseases, affect a small percentage of the population and are often associated with significant morbidity and mortality. The limited patient population makes it financially challenging for pharmaceutical companies to invest in developing new drugs specifically for these conditions, creating an urgent need for alternative therapeutic approaches.

In this thesis, we will explore the concept of drug repurposing for rare genetic disorders, focusing on the potential benefits, challenges, and implications of this approach. By repurposing existing drugs, researchers can leverage the wealth of knowledge and clinical evidence already available for these compounds, accelerating the drug discovery process and potentially bringing new treatments to market more quickly. However, there are also limitations and complexities associated with drug repurposing, including issues related to intellectual property, regulatory approval, and clinical trial design.

Chapter 1: Introduction
1.1 Introduction
1.2 Background of study
1.3 Problem Statement
1.4 Objective of study
1.5 Limitation of study
1.6 Scope of study
1.7 Significance of study
1.8 Structure of the Thesis
1.9 Definition of terms

Chapter 2: Literature Review
2.1 Overview of rare genetic disorders
2.2 Drug development challenges for rare diseases
2.3 Drug repurposing strategies and approaches
2.4 Success stories in drug repurposing for rare genetic disorders
2.5 Challenges and limitations of drug repurposing
2.6 Regulatory considerations for repurposed drugs
2.7 Intellectual property issues in drug repurposing
2.8 Clinical trial design for repurposed drugs
2.9 Ethical considerations in drug repurposing
2.10 Future directions in drug repurposing research

Chapter 3: Research Methodology
3.1 Research design
3.2 Data collection methods
3.3 Data analysis techniques
3.4 Study population
3.5 Inclusion criteria
3.6 Exclusion criteria
3.7 Ethical considerations
3.8 Validity and reliability of findings

Chapter 4: Discussion of Findings
4.1 Analysis of data
4.2 Comparison with existing literature
4.3 Implications for drug repurposing research
4.4 Recommendations for future research
4.5 Limitations of the study
4.6 Practical implications for healthcare providers
4.7 Public health implications
4.8 Policy recommendations

Chapter 5: Conclusion and Summary
5.1 Summary of key findings
5.2 Contributions to the field
5.3 Implications for clinical practice
5.4 Recommendations for policymakers
5.5 Future research directions

Thesis Overview: Drug repurposing for rare genetic disorders

Drugs repurposing for rare genetic disorders is a novel approach that holds great promise for addressing the unmet medical needs of patients with rare genetic disorders. This thesis explores the concept of drug repurposing, highlighting its potential benefits, challenges, and implications. Through a comprehensive literature review, research methodology, and discussion of findings, this thesis aims to provide insights into the opportunities and limitations of drug repurposing for rare genetic disorders. Furthermore, this thesis seeks to contribute to the growing body of knowledge in this field and offer recommendations for future research and clinical practice.

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