Select Your Own Disease
In this course, we discussed Duchenne muscular dystrophy in extensive detail. We explored the mechanism of disease and the many different therapies that have been investigated to treat patients with DMD. However, these clinical lessons are not specific to DMD. They can be extended to the study of other diseases.
In this assignment, you will write an analytic research paper about any on-going disease research which closely relates to the disease mechanism of DMD as presented by our course. These will typically include, but are not limited to, rare genetic diseases. The objective of this research paper is for you to conduct a scholarly exploration of a novel disease. Your job is to critically analyze and evaluate the history, treatment, and current state of the field just as we have done throughout this course. Based on these scientific principles, you will compare your selected disease to DMD. Discuss your findings and support your analysis with primary sources.
Please address the following issues:
Selected disease
Why did you choose this disease?
How is it similar to DMD?
How is it different from DMD?
Disease discovery
Provide a history of the disease
Initial discovery of the disease
Description of disease
Typical diagnosis
Symptoms
Prognosis
Mechanism of disease
Public health information of disease
Descriptive epidemiology (including but not limited to):
Prevalence
Incidence
Patterns
Populations
Risk factors
Comorbidity
Health care costs
Human experience of disease
Therapies for the disease
Historical therapies
Past therapies, failed and/or successful
Current gold standard of treatment
Please describe a comprehensive, multidisciplinary care plan just as we discussed for DMD
Discuss the potential efficacy of at least two therapeutic approaches discussed in our course for the treatment of your selected disease
E.g. CRISPR/Cas9, AAV delivery, Stem Cell Therapy, devices, drugs, procedures, etc.
On-going clinical trials (at least two if there are multiple on-going trials)
Evaluation of new trials compared to older trials
Comparison to DMD
Compare and contrast the current scientific literature on DMD versus your selected disease
Evaluate current clinical research progress of proposed disease
Analyzing areas for improvement and discussing possible solutions
Future direction
Rules:
Be sure to include proper citations (6-10 citations minimum).
You can consult Pubmed and the PS121 library of research papers, as well as any other reliable resources. Please use standard APA, MLA, or Chicago formatting. This can be easily done with Google scholar.
You cannot choose BMD, DMD, MEB, WW, or other diseases discussed in our course unless you receive TA approval by the end of week 9. Failure to follow this rule will result in an automatic 0% for this assignment.
Check in with instructor. You must meet with instructor before May 10th, for a 15-minute check in. You will introduce your paper idea and discuss your direction. No final submissions are required at this point.
You may not choose diseases that have been virtually eradicated, such as smallpox.
The paper should be double-spaced, limited to 12 pages not including citations and figures.
I’ve attached the rubric below, please let me know if you need sources.
