Precision medicine approaches for heterogeneous conditions such as autism spectrum disorders (The need for a biomarker exploration phase in clinical trials - Phase 2m)

Precision medicine approaches for heterogeneous conditions such as autism spectrum disorders (The need for a biomarker exploration phase in clinical trials - Phase 2m) PDF Author: David Quentin Beversdorf
Publisher: Frontiers Media SA
ISBN: 2832515681
Category : Medical
Languages : en
Pages : 153

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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.

Precision medicine approaches for heterogeneous conditions such as autism spectrum disorders (The need for a biomarker exploration phase in clinical trials - Phase 2m)

Precision medicine approaches for heterogeneous conditions such as autism spectrum disorders (The need for a biomarker exploration phase in clinical trials - Phase 2m) PDF Author: David Quentin Beversdorf
Publisher: Frontiers Media SA
ISBN: 2832515681
Category : Medical
Languages : en
Pages : 153

Get Book Here

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.

Handbook of Biomarkers and Precision Medicine

Handbook of Biomarkers and Precision Medicine PDF Author: Claudio Carini
Publisher: CRC Press
ISBN: 0429576730
Category : Mathematics
Languages : en
Pages : 631

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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.

Biomarker Tests for Molecularly Targeted Therapies

Biomarker Tests for Molecularly Targeted Therapies PDF Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309381347
Category : Medical
Languages : en
Pages : 293

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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.

Enabling Precision Medicine

Enabling Precision Medicine PDF Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309462630
Category : Medical
Languages : en
Pages : 145

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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.

The CA3 Region of the Hippocampus: How is it? What is it for? How does it do it?

The CA3 Region of the Hippocampus: How is it? What is it for? How does it do it? PDF Author: Enrico Cherubini
Publisher: Frontiers Media SA
ISBN: 2889196313
Category : Hippocampus (Brain)
Languages : en
Pages : 167

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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.

Genomic Disorders

Genomic Disorders PDF Author: James R. Lupski
Publisher: Springer Science & Business Media
ISBN: 1597450391
Category : Medical
Languages : en
Pages : 419

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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.

Genomic Medicine

Genomic Medicine PDF Author: Dhavendra Kumar
Publisher: Oxford Monographs on Medical G
ISBN: 019989602X
Category : Medical
Languages : en
Pages : 853

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Book Description
Preceded by Genomics and clinical medicine / edited by Dhavendra Kumar. [First edition]. 2008.

Health of People, Health of Planet and Our Responsibility

Health of People, Health of Planet and Our Responsibility PDF Author: Wael Al-Delaimy
Publisher: Springer Nature
ISBN: 3030311252
Category : Medical
Languages : en
Pages : 417

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Book Description
This open access book not only describes the challenges of climate disruption, but also presents solutions. The challenges described include air pollution, climate change, extreme weather, and related health impacts that range from heat stress, vector-borne diseases, food and water insecurity and chronic diseases to malnutrition and mental well-being. The influence of humans on climate change has been established through extensive published evidence and reports. However, the connections between climate change, the health of the planet and the impact on human health have not received the same level of attention. Therefore, the global focus on the public health impacts of climate change is a relatively recent area of interest. This focus is timely since scientists have concluded that changes in climate have led to new weather extremes such as floods, storms, heat waves, droughts and fires, in turn leading to more than 600,000 deaths and the displacement of nearly 4 billion people in the last 20 years. Previous work on the health impacts of climate change was limited mostly to epidemiologic approaches and outcomes and focused less on multidisciplinary, multi-faceted collaborations between physical scientists, public health researchers and policy makers. Further, there was little attention paid to faith-based and ethical approaches to the problem. The solutions and actions we explore in this book engage diverse sectors of civil society, faith leadership, and political leadership, all oriented by ethics, advocacy, and policy with a special focus on poor and vulnerable populations. The book highlights areas we think will resonate broadly with the public, faith leaders, researchers and students across disciplines including the humanities, and policy makers.

Handbook of hair in health and disease

Handbook of hair in health and disease PDF Author: Victor R. Preedy
Publisher: Springer
ISBN: 9086867286
Category : Science
Languages : en
Pages : 477

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Book Description
Hair is a major component of the body's tissue system that contributes to the individual's make up and confers a large degree of personal identity. Apart from its visible façade, hair also has a functional role. It has an unique structure and complex molecular development. The very nature of hair makes it a suitable marker for the prognosis of disease. Hair can also be used to screen for toxins and changes in the diet. However, there are currently no suitable publications available that describe hair in a rational scientific context. This handbook provides an academic approach to hair in health and disease. Divided into five sections the Handbook of Hair in Health and Disease provides an insight into hair growth and loss, molecular and cellular biology of hair, dietary toxicity and pathological history, diseases and treatments of hair, as well as shampoos and conditioners. Unique features of each chapter in this volume include relevant and useful 'Key facts' which highlight interesting or important findings of the specific subjects and 'Summary points' that will give a clear overview of the subjects treated in each chapter. The Handbook of Hair in Health and Disease will be essential to a variety of users, such as trichologists, doctors and nurses and all those interested or working within the area of hair health. This includes nutritionists and dieticians, scientific beauticians, health workers and practitioners, college and university lecturers and undergraduate and graduate students.

The Science of Health Disparities Research

The Science of Health Disparities Research PDF Author: Irene Dankwa-Mullan
Publisher: John Wiley & Sons
ISBN: 1119374812
Category : Science
Languages : en
Pages : 548

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Book Description
Integrates the various disciplines of the science of health disparities in one comprehensive volume The Science of Health Disparities Research is an indispensable source of up-to-date information on clinical and translational health disparities science. Building upon the advances in health disparities research over the past decade, this authoritative volume informs policies and practices addressing the diseases, disorders, and gaps in health outcomes that are more prevalent in minority populations and socially disadvantaged communities. Contributions by recognized scholars and leaders in the field—featuring contemporary research, conceptual models, and a broad range of scientific perspectives—provide an interdisciplinary approach to reducing inequalities in population health, encouraging community engagement in the research process, and promoting social justice. In-depth chapters help readers better understand the specifics of minority health and health disparities while demonstrating the importance of advancing theory, refining measurement, improving investigative methods, and diversifying scientific research. In 26 chapters, the book examines topics including the etiology of health disparities research, the determinants of population health, research ethics, and research in African American, Asians, Latino, American Indian, and other vulnerable populations. Providing a unified framework on the principles and applications of the science of health disparities research, this important volume: Defines the field of health disparities science and suggests new directions in scholarship and research Explains basic definitions, principles, and concepts for identifying, understanding and addressing health disparities Provides guidance on both conducting health disparities research and translating the results Examines how social, historical and contemporary injustices may influence the health of racial and ethnic minorities Illustrates the increasing national and global importance of addressing health disparities Discusses population health training, capacity-building, and the transdisciplinary tools needed to advance health equity A significant contribution to the field, The Science of Health Disparities Research is an essential resource for students and basic and clinical researchers in genetics, population genetics, and public health, health care policymakers, and epidemiologists, medical students, and clinicians, particularly those working with minority, vulnerable, or underserved populations.