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Drug development for rare diseases

A disease is defined as rare if the prevalence is fewer than 200,000 in the United States. It is estimated that there are more than 7,000 rare diseases, which collectively affect 30 million Americans or 10% of the US population. This diverse and complex disease area poses challenges for patients, caregivers, regulators, drug developers, and other stakeholders. This book is proposed to give an overview of the common issues facing rare disease drug developers, summarize challenges specific to clinical development in small populations, discuss drug development strategies in the evolving regulatory environment, explain generation and utilization of different data and evidence

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Drug delivery trends ; Vol. 3 : Expectations and realities of multifunctional drug delivery systems

Examines a drift in the pharmaceutical field across the wide range of dosage forms, drug delivery systems (micro and nanoparticulate), at the regulatory front and on new types of therapies in the market. This book covers the challenges on drug delivery systems in terms of preclinical and current ways of determining quality and the options to solve the challenges associated with this. Most small-medium scale industries and academics struggle with initial regulatory challenges so a detailed discussion on regulatory trend covers the necessary basic understanding of regulatory procedures and provides the required guidance.

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Drug Delivery Systems for Metabolic Disorders

Covers recent developments in advanced drug delivery systems in various metabolic disorders, including disturbances in protein, lipid, carbohydrate and hormone metabolism and lysosomal and mitochondrial disorders. It provides a brief introduction to metabolic disorders, along with a focus on the current landscape and trends in understanding disease pathology using different in vitro and in vivo models required for clinical applications and developments of new therapeutics.

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Drug delivery aspects : Expectations and realities of multifunctional drug delivery systems ; Vol.4 : Expectations and realities of multifunctional drug delivery systems

Reviews additional features of drug delivery systems, along with the standard formulation development, like preclinical testing, conversion into solid dosage forms, roles of excipients and polymers used on stability and sterile processing. There is a focus on formulation engineering and related large scale (GMP) manufacturing, regulatory, and functional aspects of drug delivery systems. A detailed discussion on biologics and vaccines gives insights to readers on new developments in this direction.

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Drug delivery : An integrated clinical and engineering approach

Offers a much needed comprehensive overview and patient-oriented approach for enhanced drug delivery optimization and advancement. Starting with an introduction to the subject and pharmacokinetics, it explores advances for such topics as oral, gastroretentive, intravitreal, and intrathecal drug delivery, as well as insulin delivery, gene delivery, and biomaterials-based delivery systems. It also describes drug delivery in cancer, cardiac, infectious diseases, airway diseases, and obstetrics and gynecology applications.

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Drug and Alcohol Abuse : A Clinical Guide to Diagnosis and Treatment

Gives professionals and students across the health and mental health disciplines a working knowledge of drug-related pathology, beginning with pharmacology, drug mechanisms, and genetic factors. This title features clinical/emergency orientation suited to both chronic misuse and acute situations.

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Double Balloon Endoscopy : Theory and Practice

The chapters of this book describe the principles, insertion technique, and clinical applications of double-balloon endoscopy, which will become the gold standard of enteroscopy and may well eliminate the need for intraoperative enteroscopy in many patients.

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Dose Finding in Drug Development

When you go to the pharmacy and fill a prescription, have you ever wondered if the dose of the medication is right for you? Can the dose be too low so that the drug will not work? This book answers some of these questions, and introduces the drug development process, the design and analysis of clinical trials.

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Doppler Ultrasound in Obstetrics and Gynecology

Doppler Ultrasound in Obstetrics & Gynecology encompasses the full spectrum of clinical applications of Doppler ultrasound for the practicing obstetrician-gynecologist, including the latest advances in 3D and color Doppler and the newest techniques in 4D fetal echocardiography. Written by preeminent experts in the field, the book covers the basic and physical principles of Doppler ultrasound; the use of Doppler for fetal examination, including fetal cerebral circulation; Doppler echocardiography of the fetal heart; and the use of Doppler for postdated pregnancy and in cases of multiple gestation. Chapters on the use of Doppler for gynecologic investigation include ultrasound in ectopic pregnancy, for infertility, for benign disorders and for gynecologic malignancies.

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Dissecting the Molecular Anatomy of Tissue

The book provides an updated overview of molecular analysis of human tissues, and the impact this analysis has on diagnosis and prognosis of human diseases. Special emphasis is placed on human cancer and the future directions of the field. Methods of handling clinical tissue samples, including the impact of handling on subsequent molecular analysis, are also discussed. In addition, detailed protocols for molecular analysis of DNA, RNA and protein, with special emphasis on molecular analysis of highly complex human tissue samples containing mixtures of cell populations, are provided.

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Disinfection of root canal systems : The Ttreatment of apical periodontitis

An evidence-based manual that describes root canal anatomy, the endodontic biofilm, and the role of disinfection before presenting the most up-to-date methods of irrigation and disinfection. Individual chapters are devoted to each method, such as positive pressure irrigation, apical negative pressure irrigation, sonic activation, photodynamic therapy, laser technology, and ozonization and electrochemical activation. Clinical photographs throughout show proper irrigation and disinfection techniques.

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Diseases of the Heart, Chest & Breast : Diagnostic Imaging and Interventional Techniques

This book deals with imaging of diseases of heart, chest and breast. These fields have substantially advanced during the last few years, driven by both clinical developments and advances in imaging technology.لإheir chapters are disease-oriented and cover all the relevant imaging modalities, including standard radiography, CT, nuclear medicine with PET, ultrasound and magnetic resonance imaging, as well as imaging-guided interventions. As a result, this book presents a comprehensive review of current knowledge in imaging of the heart and chest , as well as thoracic interventions and a selection of "hot topics" of breast imaging.

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Diseases of the chest, heart and vascular system 2025-2028 : Diagnostic imaging

Offers an essential overview of chest, heart and vascular system imaging. Over the last few years, there have been considerable advances in this area, driven by both clinical and technological developments. Written by leading international experts and teachers, the chapters are disease-oriented and cover all relevant imaging modalities, with a focus on magnetic resonance imaging and computed tomography. IDKD books are rewritten (not merely updated) every four years, which means they offer a comprehensive review of the state-of-the-art in imaging. The book is clearly structured and features learning objectives, abstracts, subheadings, tables and take-home points, supported by design elements to help readers navigate the text.

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Diseases of the Brain, Head and Neck, Spine 2020–2023 : Diagnostic Imaging

This book offers an essential overview of brain, head and neck, and spine imaging. Over the last few years, there have been considerable advances in this area, driven by both clinical and technological developments.

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Diseases of the Brain, Head & Neck, Spine : Diagnostic Imaging and Interventional Techniques 40th International Diagnostic Course in Davos (IDKD) Davos, March 30–April 4, 2008

This book deals with imaging of diseases of brain, head and neck, and spine. These fields have substantially advanced during the last few years, driven by both clinical developments and advances in imaging technology.

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Diseases of the Abdomen and Pelvis 2018-2021 : Diagnostic Imaging - IDKD Book

This open access book deals with imaging of the abdomen and pelvis, an area that has seen considerable advances over the past several years, driven by clinical as well as technological developments. The respective chapters, written by internationally respected experts in their fields, focus on imaging diagnosis and interventional therapies in abdominal and pelvic disease; they cover all relevant imaging modalities, including magnetic resonance imaging, computed tomography, and positron emission tomography. As such, the book offers a comprehensive review of the state of the art in imaging of the abdomen and pelvis. It will be of interest to general radiologists, radiology residents, interventional radiologists, and clinicians from other specialties who want to update their knowledge in this area.

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Diseases and conditions in dentistry : An evidence-based reference

One-stop guide for dentistry clinicians to keep at their side. Provides a quick reference for the busy clinician covering diseases and conditions in endodontics, periodontics, prosthodontics and restorative dentistry. Offers identically formatted chapters following the same clear and concise layout with detailed clinical cases and evidence-based discussions. Features a companion website with additional clinical photographs, radiographs, and case notes

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DiPiro’s pharmacotherapy : A pathophysiologic approach

It is this in-depth knowledge of pharmacotherapy, combined with the clinical experience gained through postgraduate training in direct patient care practice, that allows the pharmacist to be the "medication therapy expert" on the healthcare team and to proactively address medication-related problems.It is provides knowledge necessary to prevent, identify, and manage disease and medication-related problems

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Dipeptidyl Aminopeptidases : Basic Science and Clinical Applications

Dipeptidyl Aminopeptidases exert a potent modulatory role at an interface between immune mechanisms, metabolic responses, and neuroendocrine pathways. Experimental models and clinical studies addressing the role of these enzymes and the effect of specific inhibitors pave the way to novel therapeutic concepts in immunology, rheumatology, oncology, reproductive medicine and diabetes. Leading experts in the field will contribute to this book, which will present a state-of-the-art view on these enzymes at a time when our understanding of their function is growing ever more rapidly and therapeutic options have become imminent. The sections of the book will focus on various topics including DP IV and related enzymes in: expression and function, metabolic disorders, immune mechanisms and immune disorders, neuronal diseases and cancer, and related drug development.

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Dilated Cardiomyopathy : From Genetics to Clinical Management

This book presents a comprehensive overview of dilated cardiomyopathy, providing readers with practical guidelines for its clinical management. The first part of the book analyzes in detail the disease’s pathophysiology, its diagnostic work up as well as the prognostic stratification, and illustrates the role of genetics and gene-environment interaction. The second part presents current and future treatment options, highlighting the importance of long-term and individualized treatments and follow-up.

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