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Tailored drug therapies for rare diseases
Tailored drug therapies for rare diseases
Order ID 53003233773 Type Essay Writer Level Masters Style APA Sources/References 4 Perfect Number of Pages to Order 5-10 Pages Description/Paper Instructions
Tailored drug therapies for rare diseases
Rare diseases, also known as orphan diseases, are a group of disorders that affect a relatively small number of individuals in a given population. According to the World Health Organization (WHO), a disease is considered rare when it affects less than 1 in 2,000 people. There are estimated to be over 7,000 rare diseases, affecting more than 400 million people worldwide. Many of these rare diseases are genetic in origin and have no known cure.
Drug therapies for rare diseases have traditionally been limited due to the small patient population and the high cost of drug development. In recent years, there has been a shift towards developing tailored drug therapies for rare diseases, also known as precision medicine. Tailored drug therapies are designed to target the specific genetic or molecular abnormalities that cause a particular rare disease. By targeting the underlying cause of a rare disease, tailored drug therapies have the potential to provide more effective treatments with fewer side effects.
The development of tailored drug therapies for rare diseases typically involves a number of steps, including genetic testing, identification of a drug target, drug discovery and development, clinical trials, and regulatory approval. The first step is to identify the genetic or molecular abnormality that is responsible for the rare disease. This is often done through genetic testing, which can identify mutations in specific genes that cause the disease. Once a drug target has been identified, drug discovery and development can begin. This involves the design and synthesis of compounds that can target the specific genetic or molecular abnormality.
One of the challenges of developing tailored drug therapies for rare diseases is the small patient population. Clinical trials for rare diseases typically enroll a small number of patients, which can make it difficult to gather sufficient data to demonstrate safety and efficacy. In addition, many rare diseases have heterogeneous symptoms and manifestations, which can further complicate the design and interpretation of clinical trials.
Despite these challenges, there have been a number of successful examples of tailored drug therapies for rare diseases. One such example is Kalydeco (ivacaftor), a drug developed by Vertex Pharmaceuticals for the treatment of cystic fibrosis (CF). CF is a rare genetic disease that affects approximately 30,000 people in the United States. Kalydeco targets a specific genetic mutation that is responsible for a subset of CF cases. In clinical trials, Kalydeco demonstrated significant improvements in lung function and other key outcomes in patients with this specific mutation. Kalydeco was approved by the US Food and Drug Administration (FDA) in 2012 and has since been used to treat thousands of patients with CF.
Another example of a tailored drug therapy for a rare disease is Spin Raza (nusinersen), a drug developed by Biogen for the treatment of spinal muscular atrophy (SMA). SMA is a rare genetic disease that affects approximately 1 in 10,000 people worldwide. Spinraza targets the genetic abnormality that causes SMA by increasing the production of a protein that is critical for motor neuron function. In clinical trials, Spinraza demonstrated significant improvements in motor function and survival in patients with SMA. Spinraza was approved by the FDA in 2016 and has since been used to treat thousands of patients with SMA.
The success of these and other tailored drug therapies for rare diseases has led to increased interest and investment in precision medicine. In addition to the development of new drugs, precision medicine also includes the use of existing drugs in new ways. For example, drug repurposing involves identifying new therapeutic uses for drugs that have already been approved for other indications. This approach can be particularly useful for rare diseases, as it can help to bypass many of the early stages of drug development and reduce the time and cost of bringing new therapies to market.
Tailored drug therapies for rare diseases
RUBRIC
QUALITY OF RESPONSE NO RESPONSE POOR / UNSATISFACTORY SATISFACTORY GOOD EXCELLENT Content (worth a maximum of 50% of the total points) Zero points: Student failed to submit the final paper. 20 points out of 50: The essay illustrates poor understanding of the relevant material by failing to address or incorrectly addressing the relevant content; failing to identify or inaccurately explaining/defining key concepts/ideas; ignoring or incorrectly explaining key points/claims and the reasoning behind them; and/or incorrectly or inappropriately using terminology; and elements of the response are lacking. 30 points out of 50: The essay illustrates a rudimentary understanding of the relevant material by mentioning but not full explaining the relevant content; identifying some of the key concepts/ideas though failing to fully or accurately explain many of them; using terminology, though sometimes inaccurately or inappropriately; and/or incorporating some key claims/points but failing to explain the reasoning behind them or doing so inaccurately. Elements of the required response may also be lacking. 40 points out of 50: The essay illustrates solid understanding of the relevant material by correctly addressing most of the relevant content; identifying and explaining most of the key concepts/ideas; using correct terminology; explaining the reasoning behind most of the key points/claims; and/or where necessary or useful, substantiating some points with accurate examples. The answer is complete. 50 points: The essay illustrates exemplary understanding of the relevant material by thoroughly and correctly addressing the relevant content; identifying and explaining all of the key concepts/ideas; using correct terminology explaining the reasoning behind key points/claims and substantiating, as necessary/useful, points with several accurate and illuminating examples. No aspects of the required answer are missing. Use of Sources (worth a maximum of 20% of the total points). Zero points: Student failed to include citations and/or references. Or the student failed to submit a final paper. 5 out 20 points: Sources are seldom cited to support statements and/or format of citations are not recognizable as APA 6th Edition format. There are major errors in the formation of the references and citations. And/or there is a major reliance on highly questionable. The Student fails to provide an adequate synthesis of research collected for the paper. 10 out 20 points: References to scholarly sources are occasionally given; many statements seem unsubstantiated. Frequent errors in APA 6th Edition format, leaving the reader confused about the source of the information. There are significant errors of the formation in the references and citations. And/or there is a significant use of highly questionable sources. 15 out 20 points: Credible Scholarly sources are used effectively support claims and are, for the most part, clear and fairly represented. APA 6th Edition is used with only a few minor errors. There are minor errors in reference and/or citations. And/or there is some use of questionable sources. 20 points: Credible scholarly sources are used to give compelling evidence to support claims and are clearly and fairly represented. APA 6th Edition format is used accurately and consistently. The student uses above the maximum required references in the development of the assignment. Grammar (worth maximum of 20% of total points) Zero points: Student failed to submit the final paper. 5 points out of 20: The paper does not communicate ideas/points clearly due to inappropriate use of terminology and vague language; thoughts and sentences are disjointed or incomprehensible; organization lacking; and/or numerous grammatical, spelling/punctuation errors 10 points out 20: The paper is often unclear and difficult to follow due to some inappropriate terminology and/or vague language; ideas may be fragmented, wandering and/or repetitive; poor organization; and/or some grammatical, spelling, punctuation errors 15 points out of 20: The paper is mostly clear as a result of appropriate use of terminology and minimal vagueness; no tangents and no repetition; fairly good organization; almost perfect grammar, spelling, punctuation, and word usage. 20 points: The paper is clear, concise, and a pleasure to read as a result of appropriate and precise use of terminology; total coherence of thoughts and presentation and logical organization; and the essay is error free. 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The paper has slight errors within the paper. This can include small errors or omissions with the cover page, abstract, page number, and headers. There could be also slight formatting issues with the document spacing or the font Additionally the paper might slightly exceed or undershoot the specific number of required written pages for the assignment. 10 points: Student provides a high-caliber, formatted paper. This includes an APA 6th edition cover page, abstract, page number, headers and is double spaced in 12’ Times Roman Font. Additionally, the paper conforms to the specific number of required written pages and neither goes over or under the specified length of the paper.
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