Pub Date : 2024-11-08DOI: 10.1038/d41573-024-00183-4
Subha B. Basu
{"title":"Benchmarking recruitment rates for phase III trials","authors":"Subha B. Basu","doi":"10.1038/d41573-024-00183-4","DOIUrl":"10.1038/d41573-024-00183-4","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"23 12","pages":"887-888"},"PeriodicalIF":122.7,"publicationDate":"2024-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142598085","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-11-05DOI: 10.1038/d41573-024-00178-1
Asher Mullard
{"title":"Lundbeck pays US$2.6 billion for Longboard and its lead epilepsy drug","authors":"Asher Mullard","doi":"10.1038/d41573-024-00178-1","DOIUrl":"10.1038/d41573-024-00178-1","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"23 12","pages":"885-885"},"PeriodicalIF":122.7,"publicationDate":"2024-11-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142580386","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-11-04DOI: 10.1038/s41573-024-01059-3
Anneliene H. Jonker, Elena-Alexandra Tataru, Holm Graessner, David Dimmock, Adam Jaffe, Gareth Baynam, James Davies, Shruti Mitkus, Oxana Iliach, Rich Horgan, Erika F. Augustine, Alison Bateman-House, Anna Maria Gerdina Pasmooij, Tim Yu, Matthis Synofzik, Julie Douville, Larissa Lapteva, Philip John Brooks, Daniel O’Connor, Annemieke Aartsma-Rus, on behalf of The N-of-1 Task Force of the International Rare Diseases Research Consortium (IRDiRC)
In recent years, a small number of people with rare diseases caused by unique genetic variants have been treated with therapies developed specifically for them. This pioneering field of genetic N-of-1 therapies is evolving rapidly, giving hope for the individualized treatment of people living with very rare diseases. In this Review, we outline the concept of N-of-1 individualized therapies, focusing on genetic therapies, and illustrate advances and challenges in the field using cases for which therapies have been successfully developed. We discuss why the traditional drug development and reimbursement pathway is not fit for purpose in this field, and outline the pragmatic, regulatory and ethical challenges this poses for future access to N-of-1 therapies. Finally, we provide a roadmap for N-of-1 individualized therapy development. A very small number of people with rare diseases caused by unique genetic variants have been treated with therapies developed specifically for them, known as N-of-1 therapies. This Review discusses advances and challenges for N-of-1 therapies based on cases in which they have been successfully developed, highlights why the traditional drug development and reimbursement pathway is not fit for purpose in this field, and provides a roadmap for the development of these individualized therapies.
{"title":"The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap","authors":"Anneliene H. Jonker, Elena-Alexandra Tataru, Holm Graessner, David Dimmock, Adam Jaffe, Gareth Baynam, James Davies, Shruti Mitkus, Oxana Iliach, Rich Horgan, Erika F. Augustine, Alison Bateman-House, Anna Maria Gerdina Pasmooij, Tim Yu, Matthis Synofzik, Julie Douville, Larissa Lapteva, Philip John Brooks, Daniel O’Connor, Annemieke Aartsma-Rus, on behalf of The N-of-1 Task Force of the International Rare Diseases Research Consortium (IRDiRC)","doi":"10.1038/s41573-024-01059-3","DOIUrl":"10.1038/s41573-024-01059-3","url":null,"abstract":"In recent years, a small number of people with rare diseases caused by unique genetic variants have been treated with therapies developed specifically for them. This pioneering field of genetic N-of-1 therapies is evolving rapidly, giving hope for the individualized treatment of people living with very rare diseases. In this Review, we outline the concept of N-of-1 individualized therapies, focusing on genetic therapies, and illustrate advances and challenges in the field using cases for which therapies have been successfully developed. We discuss why the traditional drug development and reimbursement pathway is not fit for purpose in this field, and outline the pragmatic, regulatory and ethical challenges this poses for future access to N-of-1 therapies. Finally, we provide a roadmap for N-of-1 individualized therapy development. A very small number of people with rare diseases caused by unique genetic variants have been treated with therapies developed specifically for them, known as N-of-1 therapies. This Review discusses advances and challenges for N-of-1 therapies based on cases in which they have been successfully developed, highlights why the traditional drug development and reimbursement pathway is not fit for purpose in this field, and provides a roadmap for the development of these individualized therapies.","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"24 1","pages":"40-56"},"PeriodicalIF":122.7,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142574386","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-11-01DOI: 10.1038/d41573-024-00177-2
Asher Mullard
Lotte Bjerre Knudsen, chief scientific advisor in research and early development at Novo Nordisk, discusses the past and future of GLP-1s and related anti-obesity drugs. Lotte Bjerre Knudsen, chief scientific advisor in research and early development at Novo Nordisk, discusses the past and future of GLP-1s and related anti-obesity drugs.
{"title":"How GLP-1 went from being a hard-to-handle hormone to a blockbuster success","authors":"Asher Mullard","doi":"10.1038/d41573-024-00177-2","DOIUrl":"10.1038/d41573-024-00177-2","url":null,"abstract":"Lotte Bjerre Knudsen, chief scientific advisor in research and early development at Novo Nordisk, discusses the past and future of GLP-1s and related anti-obesity drugs. Lotte Bjerre Knudsen, chief scientific advisor in research and early development at Novo Nordisk, discusses the past and future of GLP-1s and related anti-obesity drugs.","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"23 12","pages":"889-890"},"PeriodicalIF":122.7,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142561986","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-11-01DOI: 10.1038/d41573-024-00180-7
Asher Mullard
{"title":"Anti-tau antibody stumbles in phase II Alzheimer trial","authors":"Asher Mullard","doi":"10.1038/d41573-024-00180-7","DOIUrl":"10.1038/d41573-024-00180-7","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"23 12","pages":"883-883"},"PeriodicalIF":122.7,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142562176","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-10-30DOI: 10.1038/d41573-024-00176-3
Asher Mullard
{"title":"FDA approves PI3Kα inhibitor that does double duty as a degrader","authors":"Asher Mullard","doi":"10.1038/d41573-024-00176-3","DOIUrl":"10.1038/d41573-024-00176-3","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"23 12","pages":"885-885"},"PeriodicalIF":122.7,"publicationDate":"2024-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142541944","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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