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Author: David Quentin Beversdorf Publisher: Frontiers Media SA ISBN: 2832515681 Category : Medical Languages : en Pages : 153
Book Description
Many therapeutic interventions for autism spectrum disorder fail when they are examined in a clinical trial. Frequently, there is a subset of patients that responds very well to the intervention, while others do not, and the overall result does not yield a positive result. As autism spectrum disorder is highly heterogeneous in its underlying genetics and other etiological risk factors, as well as its heterogeneous phenotypic manifestation, this variability in response to any specific treatment is not entirely surprising. However, it remains a challenge to meaningfully subtype this heterogeneity for targeted treatment. The purpose of this research topic is to solicit articles that address the heterogeneity in autism spectrum disorder in a manner that may meaningfully contribute to targeted treatment approaches. Studies that address the heterogeneity of autism that could theoretically lead to targeted treatment, and studies that more directly address the use of a marker in association with response to a treatment, are both aspects that will contribute to this purpose. It is hoped that this Research Topic will yield articles that can help advance the field towards precision medicine in autism spectrum disorders. Manuscripts that contribute to the specification of the heterogeneity of autism spectrum disorder in a manner that could theoretically lead to targeted treatment would be appropriate for this research topic. Additionally, articles that utilize subtyping in relation to response to treatment would be appropriate for this research topic.
Author: David Quentin Beversdorf Publisher: Frontiers Media SA ISBN: 2832515681 Category : Medical Languages : en Pages : 153
Book Description
Many therapeutic interventions for autism spectrum disorder fail when they are examined in a clinical trial. Frequently, there is a subset of patients that responds very well to the intervention, while others do not, and the overall result does not yield a positive result. As autism spectrum disorder is highly heterogeneous in its underlying genetics and other etiological risk factors, as well as its heterogeneous phenotypic manifestation, this variability in response to any specific treatment is not entirely surprising. However, it remains a challenge to meaningfully subtype this heterogeneity for targeted treatment. The purpose of this research topic is to solicit articles that address the heterogeneity in autism spectrum disorder in a manner that may meaningfully contribute to targeted treatment approaches. Studies that address the heterogeneity of autism that could theoretically lead to targeted treatment, and studies that more directly address the use of a marker in association with response to a treatment, are both aspects that will contribute to this purpose. It is hoped that this Research Topic will yield articles that can help advance the field towards precision medicine in autism spectrum disorders. Manuscripts that contribute to the specification of the heterogeneity of autism spectrum disorder in a manner that could theoretically lead to targeted treatment would be appropriate for this research topic. Additionally, articles that utilize subtyping in relation to response to treatment would be appropriate for this research topic.
Author: Claudio Carini Publisher: CRC Press ISBN: 0429576730 Category : Mathematics Languages : en Pages : 631
Book Description
"The field of Biomarkers and Precision Medicine in drug development is rapidly evolving and this book presents a snapshot of exciting new approaches. By presenting a wide range of biomarker applications, discussed by knowledgeable and experienced scientists, readers will develop an appreciation of the scope and breadth of biomarker knowledge and find examples that will help them in their own work." -Maria Freire, Foundation for the National Institutes of Health Handbook of Biomarkers and Precision Medicine provides comprehensive insights into biomarker discovery and development which has driven the new era of Precision Medicine. A wide variety of renowned experts from government, academia, teaching hospitals, biotechnology and pharmaceutical companies share best practices, examples and exciting new developments. The handbook aims to provide in-depth knowledge to research scientists, students and decision makers engaged in Biomarker and Precision Medicine-centric drug development. Features: Detailed insights into biomarker discovery, validation and diagnostic development with implementation strategies Lessons-learned from successful Precision Medicine case studies A variety of exciting and emerging biomarker technologies The next frontiers and future challenges of biomarkers in Precision Medicine Claudio Carini, Mark Fidock and Alain van Gool are internationally recognized as scientific leaders in Biomarkers and Precision Medicine. They have worked for decades in academia and pharmaceutical industry in EU, USA and Asia. Currently, Dr. Carini is Honorary Faculty at Kings’s College School of Medicine, London, UK. Dr. Fidock is Vice President of Precision Medicine Laboratories at AstraZeneca, Cambridge, UK. Prof.dr. van Gool is Head Translational Metabolic Laboratory at Radboud university medical school, Nijmegen, NL.
Author: National Academies of Sciences, Engineering, and Medicine Publisher: National Academies Press ISBN: 0309381347 Category : Medical Languages : en Pages : 293
Book Description
Every patient is unique, and the evolving field of precision medicine aims to ensure the delivery of the right treatment to the right patient at the right time. In an era of rapid advances in biomedicine and enhanced understanding of the genetic basis of disease, health care providers increasingly have access to advanced technologies that may identify molecular variations specific to an individual patient, which subsequently can be targeted for treatment. Known as biomarker tests for molecularly targeted therapies, these complex tests have the potential to enable the selection of the most beneficial treatment (and also to identify treatments that may be harmful or ineffective) for the molecular underpinnings of an individual patient's disease. Such tests are key to unlocking the promise of precision medicine. Biomarker tests for molecularly targeted therapies represent a crucial area of focus for developing methods that could later be applicable to other areas of precision medicine. The appropriate regulatory oversight of these tests is required to ensure that they are accurate, reliable, properly validated, and appropriately implemented in clinical practice. Moreover, common evidentiary standards for assessing the beneficial impact of biomarker-guided therapy selection on patient outcomes, as well as the effective collection and sharing of information related to those outcomes, are urgently needed to better inform clinical decision making. Biomarker Tests of Molecularly Targeted Therapies examines opportunities for and challenges to the use of biomarker tests to select optimal therapy and offers recommendations to accelerate progress in this field. This report explores regulatory issues, reimbursement issues, and clinical practice issues related to the clinical development and use of biomarker tests for targeting therapies to patients. Properly validated, appropriately implemented biomarker tests hold the potential to enhance patient care and improve outcomes, and therefore addressing the challenges facing such tests is critical.
Author: National Academies of Sciences, Engineering, and Medicine Publisher: National Academies Press ISBN: 0309462630 Category : Medical Languages : en Pages : 145
Book Description
Those involved in the drug development process face challenges of efficiency and overall sustainability due in part to high research costs, lengthy development timelines, and late-stage drug failures. Novel clinical trial designs that enroll participants based on their genetics represent a potentially disruptive change that could improve patient outcomes, reduce costs associated with drug development, and further realize the goals of precision medicine. On March 8, 2017, the Forum on Drug Discovery, Development, and Translation and the Roundtable on Genomics and Precision Health of the National Academies of Sciences, Engineering, and Medicine hosted the workshop Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development. Participants examined successes, challenges, and possible best practices for effectively using genetic information in the design and implementation of clinical trials to support the development of precision medicines, including exploring the potential advantages and disadvantages of such trials across a variety of disease areas. This publication summarizes the presentations and discussions from the workshop.
Author: Enrico Cherubini Publisher: Frontiers Media SA ISBN: 2889196313 Category : Hippocampus (Brain) Languages : en Pages : 167
Book Description
The CA3 hippocampal region receives information from the entorhinal cortex either directly from the perforant path or indirectly from the dentate gyrus via the mossy fibers (MFs). According to their specific targets (principal/mossy cells or interneurons), MFs terminate with large boutons or small filopodial extensions, respectively. MF-CA3 synapses are characterized by a low probability of release and pronounced frequency-dependent facilitation. In addition MF terminals are endowed with mGluRs that regulate their own release. We will describe the intrinsic membrane properties of pyramidal cells, which can sometimes fire in bursts, together with the geometry of their dendritic arborization. The single layer of pyramidal cells is quite distinct from the six-layered neocortical arrangement. The resulting aligned dendrites provides the substrate for laminated excitatory inputs. They also underlie a precise, diversity of inhibitory control which we will also describe in detail. The CA3 region has an especially rich internal connectivity, with recurrent excitatory and inhibitory loops. In recent years both in vivo and in vitro studies have allowed to better understand functional properties of the CA3 auto-associative network and its role in information processing. This circuit is implicated in encoding spatial representations and episodic memories. It generates physiological population synchronies, including gamma, theta and sharp-waves that are presumed to associate firing in selected assemblies of cells in different behavioral conditions. The CA3 region is susceptible to neurodegeneration during aging and after stresses such as infection or injury. Loss of some CA3 neurones has striking effects on mossy fiber inputs and can facilitate the generation of pathologic synchrony within the CA3 micro-circuit. The aim of this special topic is to bring together experts on the cellular and molecular mechanisms regulating the wiring properties of the CA3 hippocampal microcircuit in both physiological and pathological conditions, synaptic plasticity, behavior and cognition.We will particularly emphasize the dual glutamatergic and GABAergic phenotype of MF-CA3 synapses at early developmental stages and the steps that regulate the integration of newly generated neurons into the adult dentate gyrus-CA3 circuit.
Author: J. Gavin Bremner Publisher: Wiley-Blackwell ISBN: 9780631184669 Category : Psychology Languages : en Pages : 336
Book Description
Assuming little or no prior knowledge of the subject, Infancy presents a complete picture of our current understanding of the development of infants, their knowledge and understanding of the world from birth or before to their second birthday.
Author: James R. Lupski Publisher: Springer Science & Business Media ISBN: 1597450391 Category : Medical Languages : en Pages : 419
Book Description
A grand summary and synthesis of the tremendous amount of data now available in the post genomic era on the structural features, architecture, and evolution of the human genome. The authors demonstrate how such architectural features may be important to both evolution and to explaining the susceptibility to those DNA rearrangements associated with disease. Technologies to assay for such structural variation of the human genome and to model genomic disorders in mice are also presented. Two appendices detail the genomic disorders, providing genomic features at the locus undergoing rearrangement, their clinical features, and frequency of detection.
Author: Michelle Aldridge Publisher: Multilingual Matters ISBN: 9781853593161 Category : Language Arts & Disciplines Languages : en Pages : 236
Book Description
Comprises 17 papers presented at the Child Language Seminar, Bangor 1994, with contributions in areas as diverse as bilingual development, phonological disorders, sign language development, and the language of Down's syndrome children.
Author: Alan Slater Publisher: Psychology Press ISBN: 9780863778513 Category : Psychology Languages : en Pages : 452
Book Description
The aim of this book is both to reflect current knowledge of perceptual development and to point to some of the many questions that remain unanswered. The study of perceptual development is now a sophisticated science. The majority of the chapters tell a fascinating detective story: the way in which infants perceive and understand the world as they develop. Each of the major sections is prefaced by introductory comments, and the book will be useful for advanced undergraduates, postgraduates, researchers, and other professionals who have an interest in early perceptual development and in infancy in general.
Author: David Quentin Beversdorf
Author: David Quentin Beversdorf
Author: Claudio Carini
Author: National Academies of Sciences, Engineering, and Medicine
Author: National Academies of Sciences, Engineering, and Medicine
Author: Enrico Cherubini
Author: J. Gavin Bremner
Author: James R. Lupski
Author: Dhavendra Kumar
Author: Michelle Aldridge
Author: Alan Slater