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JAK inhibition combats adverse skin reaction 抑制 JAK 可消除皮肤不良反应
IF 122.7 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2024-11-05 DOI: 10.1038/d41573-024-00181-6
Sarah Crunkhorn
{"title":"JAK inhibition combats adverse skin reaction","authors":"Sarah Crunkhorn","doi":"10.1038/d41573-024-00181-6","DOIUrl":"10.1038/d41573-024-00181-6","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"23 12","pages":"897-897"},"PeriodicalIF":122.7,"publicationDate":"2024-11-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142580259","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}
引用次数: 0
Lundbeck pays US$2.6 billion for Longboard and its lead epilepsy drug 灵北斥资 26 亿美元收购 Longboard 及其主要癫痫药物
IF 122.7 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2024-11-05 DOI: 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}
引用次数: 0
The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap N-of-1疗法的最新进展和IRDiRC N-of-1开发路线图
IF 122.7 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2024-11-04 DOI: 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)
{"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}
引用次数: 0
Bivalent molecules unmute silenced genes 双价分子能解除沉默基因的哑音
IF 122.7 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2024-11-01 DOI: 10.1038/d41573-024-00179-0
Alex Eccleston
{"title":"Bivalent molecules unmute silenced genes","authors":"Alex Eccleston","doi":"10.1038/d41573-024-00179-0","DOIUrl":"10.1038/d41573-024-00179-0","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"23 12","pages":"896-896"},"PeriodicalIF":122.7,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142561798","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}
引用次数: 0
How GLP-1 went from being a hard-to-handle hormone to a blockbuster success GLP-1 如何从一种难以驾驭的激素变成成功的大片
IF 122.7 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2024-11-01 DOI: 10.1038/d41573-024-00177-2
Asher Mullard
{"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}
引用次数: 0
Anti-tau antibody stumbles in phase II Alzheimer trial 抗 tau 抗体在阿尔茨海默症二期试验中磕磕绊绊
IF 122.7 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2024-11-01 DOI: 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}
引用次数: 0
FDA approves PI3Kα inhibitor that does double duty as a degrader 美国 FDA 批准兼具降解作用的 PI3Kα 抑制剂
IF 122.7 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2024-10-30 DOI: 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}
引用次数: 0
Multivalent vaccine tackles C. difficile 应对艰难梭菌的多价疫苗
IF 122.7 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2024-10-30 DOI: 10.1038/d41573-024-00174-5
Katie Kingwell
{"title":"Multivalent vaccine tackles C. difficile","authors":"Katie Kingwell","doi":"10.1038/d41573-024-00174-5","DOIUrl":"10.1038/d41573-024-00174-5","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"23 12","pages":"895-895"},"PeriodicalIF":122.7,"publicationDate":"2024-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142536941","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}
引用次数: 0
FDA approves first anti-TFPI antibody for haemophilia A and B 美国食品和药物管理局批准首个用于治疗 A 型和 B 型血友病的抗 TFPI 抗体
IF 122.7 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2024-10-30 DOI: 10.1038/d41573-024-00175-4
Asher Mullard
{"title":"FDA approves first anti-TFPI antibody for haemophilia A and B","authors":"Asher Mullard","doi":"10.1038/d41573-024-00175-4","DOIUrl":"10.1038/d41573-024-00175-4","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"23 12","pages":"884-884"},"PeriodicalIF":122.7,"publicationDate":"2024-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142541943","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}
引用次数: 0
Antibody vanquishes SARS-CoV-2 variants 抗体战胜 SARS-CoV-2 变体
IF 122.7 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2024-10-29 DOI: 10.1038/d41573-024-00173-6
Katie Kingwell
{"title":"Antibody vanquishes SARS-CoV-2 variants","authors":"Katie Kingwell","doi":"10.1038/d41573-024-00173-6","DOIUrl":"10.1038/d41573-024-00173-6","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"23 12","pages":"895-895"},"PeriodicalIF":122.7,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142520151","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}
引用次数: 0
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