{"title":"Excellence ... and Other Random Thoughts.","authors":"David L Pohl","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":74203,"journal":{"name":"Missouri medicine","volume":"122 1","pages":"11"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11827656/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143434011","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Haiti-A Story of Hope and Chaos.","authors":"W Abraham White, William L White","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":74203,"journal":{"name":"Missouri medicine","volume":"122 1","pages":"23-25"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11827654/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143434431","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"John Sappington MD: The Missouri Country Doctor Who Introduced Quinine for the Treatment of Malaria in North America.","authors":"Arthur Gale","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":74203,"journal":{"name":"Missouri medicine","volume":"122 1","pages":"26-27"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11827658/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143434341","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Life as a Rural Physician: Many Opportunities for Service and Impact.","authors":"Warren Lovinger","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":74203,"journal":{"name":"Missouri medicine","volume":"122 1","pages":"28-30"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11827659/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143434344","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Whooping Cough: No Longer Just Kid Stuff.","authors":"John C Hagan","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":74203,"journal":{"name":"Missouri medicine","volume":"122 1","pages":"4-6"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11827647/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143434442","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Triumphs, Trials, and Future Considerations in Genetic Therapies for Hereditary Neuromuscular Diseases.","authors":"W David Arnold, Kajri Majithia","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>Neuromuscular diseases include conditions that affect the spinal motor neurons, peripheral nerves, neuromuscular junctions, and muscles, and they can result from acquired and inherited causes. The number of genetic therapies targeting the inherited causes of neuromuscular diseases has surged in the last decade. This review aims to highlight the current state of genetic therapies within the framework of precision medicine, focusing on the achievements and the gaps that remain. A major emphasis is on spinal muscular atrophy, Duchenne muscular dystrophy, and amyotrophic lateral sclerosis, as these neuromuscular diseases have seen tremendous recent advancements. We will also discuss the future considerations necessary to accelerate the development of next-generation genetic therapies and enhance therapeutic outcomes for patients with neuromuscular diseases.</p>","PeriodicalId":74203,"journal":{"name":"Missouri medicine","volume":"122 1","pages":"46-52"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11827653/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143434440","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
David S McKinsey, Joel P McKinsey, Julie R McKinsey, Maithe Enriquez
{"title":"Optimizing Missouri's Preparation for the Next Pandemic: Lessons Learned from COVID-19: Part 2.","authors":"David S McKinsey, Joel P McKinsey, Julie R McKinsey, Maithe Enriquez","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":74203,"journal":{"name":"Missouri medicine","volume":"122 1","pages":"12-18"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11827655/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143434361","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kerry Grens, Judith L Weisenberg, Robin C Ryther, Harrison W Gabel
{"title":"From Serendipity to Scalability in Rare Disease Patient Collaborations.","authors":"Kerry Grens, Judith L Weisenberg, Robin C Ryther, Harrison W Gabel","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>As the rate of diagnosis for rare disease increases, so does the need to develop scalable solutions to address patient community needs. Drawing upon our experiences in rare intellectual and developmental disability research, advocacy, and treatment, we present two examples of how collaboration between patient groups, clinicians, and investigators at Washington University in St. Louis have generated invaluable resources to accelerate toward treatments. These successful partnerships serve as models for building research and clinical infrastructure for rare diseases.</p>","PeriodicalId":74203,"journal":{"name":"Missouri medicine","volume":"122 1","pages":"53-59"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11827657/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143434425","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}