[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"detail-sidebar-cat-0-en-105":3,"doc-seo-291519-105":59,"doc-detail-291519-en":130},{"code":4,"msg":5,"data":6},0,"success",[7,13,18,23,28,33,38,43,48,51,55],{"id":8,"doc_module":4,"doc_module_name":9,"category_name":10,"show_sort_weight":11,"slug":12},1,"Document","Story & Novel",90,"story-novel",{"id":14,"doc_module":4,"doc_module_name":9,"category_name":15,"show_sort_weight":16,"slug":17},2,"Literature",80,"literature",{"id":19,"doc_module":4,"doc_module_name":9,"category_name":20,"show_sort_weight":21,"slug":22},4,"Exam",70,"exam",{"id":24,"doc_module":4,"doc_module_name":9,"category_name":25,"show_sort_weight":26,"slug":27},5,"Comic",60,"comic",{"id":29,"doc_module":4,"doc_module_name":9,"category_name":30,"show_sort_weight":31,"slug":32},6,"Technology",50,"technology",{"id":34,"doc_module":4,"doc_module_name":9,"category_name":35,"show_sort_weight":36,"slug":37},7,"Healthcare",40,"healthcare",{"id":39,"doc_module":4,"doc_module_name":9,"category_name":40,"show_sort_weight":41,"slug":42},8,"Research & Report",30,"research-report",{"id":44,"doc_module":4,"doc_module_name":9,"category_name":45,"show_sort_weight":46,"slug":47},9,"Religion & Spirituality",20,"religion-spirituality",{"id":46,"doc_module":4,"doc_module_name":9,"category_name":49,"show_sort_weight":46,"slug":50},"World Cup","world-cup",{"id":52,"doc_module":4,"doc_module_name":9,"category_name":53,"show_sort_weight":52,"slug":54},10,"Lifestyle","lifestyle",{"id":56,"doc_module":4,"doc_module_name":9,"category_name":57,"show_sort_weight":24,"slug":58},19,"General","general",{"code":4,"msg":60,"data":61},"ok",{"site_id":62,"language":63,"slug":64,"title":65,"keywords":66,"description":67,"schema_data":68,"social_meta":123,"head_meta":125,"extra_data":127,"updated_unix":129},105,"en","precision-medicines-for-neurodegenerative-disorders-optimizing-clinical-development","Precision medicines for neurodegenerative disorders - optimizing clinical development","","Precision medicine enables individualized care by combining lifestyle and environmental inputs with molecular, cellular, image, and genomic profiling. Adoption is accelerating as sequencing costs decline and disease-associated genomic variants and biomarkers expand, including for neurodegeneration. Despite approvals for advanced therapies, cell and gene therapy development remains difficult because neurodegenerative diseases share overlapping mechanisms yet differ in pathophysiology, diagnosis timing, and patient risk characterization, requiring tailored, patient-centric clinical development strategies for effective trials.",{"@graph":69,"@context":122},[70,84,105],{"@type":71,"itemListElement":72},"BreadcrumbList",[73,77,79,82],{"item":74,"name":75,"@type":76,"position":8},"https://docshare.wps.com","Home","ListItem",{"item":78,"name":9,"@type":76,"position":14},"https://docshare.wps.com/document/",{"item":80,"name":40,"@type":76,"position":81},"https://docshare.wps.com/document/research-report/",3,{"item":83,"name":65,"@type":76,"position":19},"https://docshare.wps.com/document/precision-medicines-for-neurodegenerative-disorders-optimizing-clinical-development/291519/",{"url":83,"name":65,"@type":85,"image":86,"author":91,"headline":65,"publisher":94,"fileFormat":97,"inLanguage":63,"description":67,"dateModified":98,"datePublished":99,"encodingFormat":97,"isAccessibleForFree":100,"interactionStatistic":101},"DigitalDocument",{"url":87,"@type":88,"width":89,"height":90},"https://docshare.wps.com/thumbnails/precision-medicines-for-neurodegenerative-disorders-optimizing-clinical-development/291519.png","ImageObject",300,407,{"name":92,"@type":93},"Rizky","Person",{"url":74,"name":95,"@type":96},"DocShare","Organization","application/pdf","2026-10-18","2026-09-17",true,{"@type":102,"interactionType":103,"userInteractionCount":52},"InteractionCounter",{"@type":104},"ViewAction",{"@type":106,"mainEntity":107},"FAQPage",[108,114,118],{"name":109,"@type":110,"acceptedAnswer":111},"Why is precision medicine important for neurodegenerative disorders?","Question",{"text":112,"@type":113},"It uses multi-omic and genomic information to tailor treatment and disease management to the specific patient, which is crucial for heterogeneous neurodegenerative conditions.","Answer",{"name":115,"@type":110,"acceptedAnswer":116},"What makes clinical development challenging in cell and gene therapies for neurodegeneration?",{"text":117,"@type":113},"Key challenges include overlapping symptoms that complicate early differential diagnosis, limited predictive preclinical models, and timing-sensitive effects across disease stages.",{"name":119,"@type":110,"acceptedAnswer":120},"How does genetic testing affect patient selection for trials?",{"text":121,"@type":113},"Genetic testing can identify at-risk individuals, but when risks cannot be precisely characterized and diseases involve multiple genes, it may be less predictive of clinical success and adds psychological and financial impacts.","https://schema.org",{"og:url":83,"og:type":124,"og:title":65,"og:site_name":95,"og:description":67},"article",{"robots":126,"canonical":83},"index,follow",{"doc_id":128,"site_id":62},291519,1789645632,{"code":4,"msg":5,"data":131},{"doc_id":128,"user_id":132,"nickname":92,"user_avatar":133,"doc_module":4,"category_id":39,"category_name":40,"doc_title":65,"doc_description":67,"doc_content":134,"file_id":135,"file_url":136,"file_type":137,"file_size":138,"view_count":52,"is_deleted":4,"is_public":8,"is_downloadable":8,"audit_status":8,"page_count":139,"language":140,"language_code":63,"site_id":62,"html_lang":63,"table_of_contents":141,"faqs":142,"seo_title":143,"seo_description":67,"update_tm":129,"read_time":144,"preview_page_count":4},962085564807,"https://ap-avatar.wpscdn.com/davatar_6f874abed73319feea01a86fa6f0fab8","WHITE PAPER  \nPrecision medicines for neurodegenerative disorders:  \noptimizing clinical development  \nContributors  \nFrederick Derosier, DO, Vice President Rare Diseases, Advanced Therapies and Pediatrics, Fortrea Tetyana Korchak, MD, Global Lead, Strategic Delivery and Growth, Neuroscience, Fortrea  \nPrecision medicine, a new paradigm of individualized treatment, is revolutionizing healthcare. This innovative approach utilizes lifestyle and environmental factors, molecular, cellular and image profiling, as well as genomic information to enable treatment and management of disease that is specialized and unique to a given patient.  \nMomentum for the implementation of precision medicine in clinical practice is increasing. The price of exome sequencing and whole genome sequencing continues to fall while the catalog of disease-associated copy number variants or deleterious sequence variants—produced from genome-wide association studies, array comparative genomic hybridization and next-generation sequencing (including exome sequencing and whole-genome sequencing)—is rapidly growing. Leveraging access to numerous biomarkers and diagnostics, oncology has been the front-runner in the successful implementation of precision medicine approaches. Similar progress in neurology and other therapeutic areas has also been witnessed with recent advances in the discovery and development of multimodal diagnostic biomarkers. As of 2022, seven advanced therapies have been approved for neurodegenerative disease.  \nWhile these advances offer a promising outlook for patients who have few or no treatment alternatives, cell and gene therapy development continues to face many challenges inherent with studying neurodegenerative disorders. From reviewing therapeutic approaches to applying a gene  \ntherapy in a neurodegenerative disorder, this article shares key considerations for addressing the specific complexities of these diseases and optimizing clinical development to support patient-centric trials.  \nUnderstanding the challenges of neurodegenerative diseases  \nEach neurodegenerative disease has a different pathophysiology, which can be identified by elements that are common across more than one disease, such as inflammation, cell death, impaired/failure of axonal regeneration, demyelination, and/or structural and functional neuronal deficits.  \nCurrent treatment strategies target a small subset of the population and focus on symptomatic relief only, without altering disease progression. Although rapid progress  \nin the understanding of the molecular and genetic  \npathogenesis of neurodegenerative diseases has led to the development of new therapeutic approaches in an attempt to arrest or delay neurodegenerative processes, many challenges still face the field, such as:  \n• Common underlying causes and unique mechanisms of toxicity 1, 3  \nCertain pathologic features of neurodegenerative disease may be common to more than one disorder and can include abnormal accumulation of proteins, aggregation of proteins, misfolded proteins, RNA toxicity and translational products from repeat expansion within genes  \n• Timely diagnosis of neurodegenerative diseases  \nWith overlapping symptoms, it is difficult to make a differential diagnosis in the early stages of a neurodegenerative disorder. By the time symptoms are evidenced, significant damage may be present as pathological alterations typically begin long before symptoms are present. Furthermore, there is a lack of predictive preclinical models that can accurately translate favorable preclinical outcomes to the clinic  \n• Potential timing considerations for treatment administration  \nKnowing that a disease progresses over time before symptoms manifest, researchers must consider the timing and the administration of a treatment. Different cell populations may be affected along the timeline, and evidence suggests that neurons may be in an atrophic state for some time before actually dying; therefore, it may be des","cbCaicKOFciM5sFw","https://ap.wps.com/l/cbCaicKOFciM5sFw","pdf",1254778,13,"English","# Contributing to precision medicines for neurodegenerative disorders\n## Precision medicine momentum and enabling technologies\n## Challenges specific to neurodegenerative disease and development\n## Understanding the challenges of neurodegenerative diseases\n## Complexities in the application of precision medicines\n## Example: Friedreich's ataxia","[{\"question\":\"Why is precision medicine important for neurodegenerative disorders?\",\"answer\":\"It uses multi-omic and genomic information to tailor treatment and disease management to the specific patient, which is crucial for heterogeneous neurodegenerative conditions.\"},{\"question\":\"What makes clinical development challenging in cell and gene therapies for neurodegeneration?\",\"answer\":\"Key challenges include overlapping symptoms that complicate early differential diagnosis, limited predictive preclinical models, and timing-sensitive effects across disease stages.\"},{\"question\":\"How does genetic testing affect patient selection for trials?\",\"answer\":\"Genetic testing can identify at-risk individuals, but when risks cannot be precisely characterized and diseases involve multiple genes, it may be less predictive of clinical success and adds psychological and financial impacts.\"}]","Precision medicines for neurodegenerative disorders - optimizing clinical development | PDF",33]