Orphan diseases are all medical conditions that impact 0.65 to 1 out of every 1000 people, according to the World Health Organization. Since these are not financially stable and feasible, they are typically not explored for their pathophysiology or the emerging therapy alternatives. On January 28, 1983, in the USA a law known as the Orphan Drug Act was formed to promote the study, creation, and approval of medications for rare diseases. There are currently as many medications for other infectious diseases as there are for tropical infectious diseases, with 11 pharmaceuticals (4.87%) recognized as orphan drugs. Several medications having orphan status are used to treat illnesses that no longer meet the requirements for orphan designation. Some businesses also manufacture and produce orphan medications. Nuclear cloning, rational drug design, and high throughput screening are all methods used in drug discovery programs that could lead to the development of new medicines. This article discusses orphan drugs, medical orphans, rare diseases, the orphan drug act, orphan drug design, the global market scenario of orphan drugs and various databases that contain information about orphan drugs. Patients who have been ignored by for-profit medication discovery efforts have hope for the future.
{"title":"Orphan Drugs: A Thematic View","authors":"Poluru SriSatya Vani, Vani Mamillapalli, Padmalatha Kantamaneni, Raya Srikeerthi, Thirumalasetty Ruchitharani, Lanka Rajini, Chilakabathini Keziahrani","doi":"10.46610/jpdra.2023.v05i02.005","DOIUrl":"https://doi.org/10.46610/jpdra.2023.v05i02.005","url":null,"abstract":"Orphan diseases are all medical conditions that impact 0.65 to 1 out of every 1000 people, according to the World Health Organization. Since these are not financially stable and feasible, they are typically not explored for their pathophysiology or the emerging therapy alternatives. On January 28, 1983, in the USA a law known as the Orphan Drug Act was formed to promote the study, creation, and approval of medications for rare diseases. There are currently as many medications for other infectious diseases as there are for tropical infectious diseases, with 11 pharmaceuticals (4.87%) recognized as orphan drugs. Several medications having orphan status are used to treat illnesses that no longer meet the requirements for orphan designation. Some businesses also manufacture and produce orphan medications. Nuclear cloning, rational drug design, and high throughput screening are all methods used in drug discovery programs that could lead to the development of new medicines. This article discusses orphan drugs, medical orphans, rare diseases, the orphan drug act, orphan drug design, the global market scenario of orphan drugs and various databases that contain information about orphan drugs. Patients who have been ignored by for-profit medication discovery efforts have hope for the future.","PeriodicalId":483910,"journal":{"name":"Journal of Pharma and Drug Regulatory Affairs","volume":"29 6","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-11-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135819920","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-10-13DOI: 10.46610/jpdra.2023.v05i02.002
Vivek kumar Tiwari, Bairi Harika
Technology is used in proteomics to count and estimate how many proteins are present in each cell, tissue, and organism. It aids in the identification of an organism's proteins and the comprehension of the structure and functions of a particular protein, complementing other "omics" technologies like genomics and transcriptomics. Proteomics is fundamentally complicated because it entails categorizing and examining all of the protein signatures in a genome. Mass spectrometry is the foundation of contemporary proteomics, with LC-MS-MS and MALDI-TOF/TOF being widely utilized devices. However, finding biomarkers is still a challenge in proteomics due to their entanglement and dynamic nature. Consequently, using a proteomics technique in conjunction with genomics and bioinformatics will help to interpret the data about biological systems and how illness alters them. However, most studies have only examined a small portion of the blood proteins. This review emphasizes the several types of proteomics, the available approaches, and their applications in numerous research fields.
{"title":"Applications of Proteomics in Human Medicine","authors":"Vivek kumar Tiwari, Bairi Harika","doi":"10.46610/jpdra.2023.v05i02.002","DOIUrl":"https://doi.org/10.46610/jpdra.2023.v05i02.002","url":null,"abstract":"Technology is used in proteomics to count and estimate how many proteins are present in each cell, tissue, and organism. It aids in the identification of an organism's proteins and the comprehension of the structure and functions of a particular protein, complementing other \"omics\" technologies like genomics and transcriptomics. Proteomics is fundamentally complicated because it entails categorizing and examining all of the protein signatures in a genome. Mass spectrometry is the foundation of contemporary proteomics, with LC-MS-MS and MALDI-TOF/TOF being widely utilized devices. However, finding biomarkers is still a challenge in proteomics due to their entanglement and dynamic nature. Consequently, using a proteomics technique in conjunction with genomics and bioinformatics will help to interpret the data about biological systems and how illness alters them. However, most studies have only examined a small portion of the blood proteins. This review emphasizes the several types of proteomics, the available approaches, and their applications in numerous research fields.","PeriodicalId":483910,"journal":{"name":"Journal of Pharma and Drug Regulatory Affairs","volume":"15 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135918401","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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