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New treatments for MND: The future of high throughput drug screening
Introduction to MND/ALS
Clinical presentation and progression
Historical context and evolution of scientific understanding
Epidemiology and impact on public health
Pathophysiology and Genetics
Loss of motor neurons in brain and spinal cord
Genetic mutations linked to familial MND
Differences between familial and sporadic cases
Risk factors including environmental and viral influences
Challenges in MND Research and Treatment
Disease heterogeneity and variability in patient subtypes
Limitations of animal models and traditional drug screening
Overview of clinical trials outcomes and difficulties
Innovative Research Approaches
Use of induced pluripotent stem cells (iPSCs) derived from patient skin biopsies
Differentiation of iPSCs into motor neurons for disease modeling
High-throughput robotic drug screening platforms
Real-time imaging and automated analysis of motor neuron survival
Personalized Medicine in MND
Molecular profiling and subtyping of patients for targeted therapy
Repurposing existing drugs for potential new treatments
Development of complex brain organoid models ("brain chips") for advanced studies
Community Engagement and Funding
Impact of global awareness campaigns on research funding
Importance of patient participation in research studies
Fundraising initiatives supporting ongoing MND research
What's in the course?
- Comprehensive Understanding of MND: Gain a clear and detailed knowledge of the molecular and clinical aspects of Motor Neuron Disease, including its genetic causes, risk factors, and disease progression.
- Exposure to Advanced Research Techniques: Learn about cutting-edge methods such as patient-derived stem cell models, high-throughput drug screening, and molecular profiling, which are shaping modern therapeutic development.
- Insight into Personalized Medicine and Drug Discovery: Understand how personalized approaches and innovative technologies are used to identify effective treatments, improving prospects for tailored therapies in MND.
Beginner
N/A
Yes
What You'll Learn
Understand the clinical and molecular characteristics of MND.
Recognize the genetic and environmental factors contributing to disease risk.
Identify challenges faced in developing effective treatments for MND.
Explain the process of generating patient-derived motor neurons for research.
Describe high-throughput drug screening methods and their application in MND.
Appreciate the role of personalized medicine in improving therapeutic outcomes.
Acknowledge the importance of community support and funding in advancing research.
Curriculum
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What's in the course?
- Comprehensive Understanding of MND: Gain a clear and detailed knowledge of the molecular and clinical aspects of Motor Neuron Disease, including its genetic causes, risk factors, and disease progression.
- Exposure to Advanced Research Techniques: Learn about cutting-edge methods such as patient-derived stem cell models, high-throughput drug screening, and molecular profiling, which are shaping modern therapeutic development.
- Insight into Personalized Medicine and Drug Discovery: Understand how personalized approaches and innovative technologies are used to identify effective treatments, improving prospects for tailored therapies in MND.