Real-world experience with CFTR modulator therapy

Real-world experience with CFTR modulator therapy PDF Author: Burkhard Tümmler
Publisher: Frontiers Media SA
ISBN: 2832540244
Category : Science
Languages : en
Pages : 151

Book Description


The Cystic Fibrosis Transmembrane Conductance Regulator

The Cystic Fibrosis Transmembrane Conductance Regulator PDF Author: Kevin L. Kirk
Publisher: Springer Science & Business Media
ISBN: 9780306478376
Category : Science
Languages : en
Pages : 236

Book Description
The CFTR chloride channel is one of the most well studied transport proteins in biology. Yet there remain many mysteries about the functional properties and biological roles of this ABC transporter. The Cystic Fibrosis Transmembrane Conductance Regulator addresses a select series of `hot' topics that relate to the function of CFTR, and the links between CFTR dysfunction and human disease (i.e., cystic fibrosis). The timeliness of these topics distinguishes this collection from previous volumes of this type. Given the general interest in CFTR, this collection will appeal to a broad readership with interests in CFTR, cystic fibrosis, ion channels and ABC transporters.

Advances in Cystic Fibrosis, An Issue of Clinics in Chest Medicine, E-Book

Advances in Cystic Fibrosis, An Issue of Clinics in Chest Medicine, E-Book PDF Author: Clemente J. Britto
Publisher: Elsevier Health Sciences
ISBN: 0323849628
Category : Medical
Languages : en
Pages : 281

Book Description
In this issue of Clinics in Chest Medicine, guest editors Drs. Clemente J. Britto and Jennifer L. Taylor-Cousar bring their considerable expertise to Advances in Cystic Fibrosis. Top experts in the field cover key topics such as genetics of cystic fibrosis (CF): clinical implications; innate and adaptive immunity in CF; novel applications of biomarkers and personalized medicine to define the spectrum of CF; emerging approaches to understand CF pathogenesis; and more. - Contains 18 relevant, practice-oriented topics including novel approaches to multidrug resistant infections in CF; clinical outcomes of highly-effective modulator therapy; nontuberculous mycobacterial infections in CF; management of mental health in CF; sexual and reproductive health in CF; nutritional management in CF; transitions of care in CF; and more. - Provides in-depth clinical reviews on advances in cystic fibrosis, offering actionable insights for clinical practice. - Presents the latest information on this timely, focused topic under the leadership of experienced editors in the field. Authors synthesize and distill the latest research and practice guidelines to create clinically significant, topic-based reviews.

Cystic Fibrosis

Cystic Fibrosis PDF Author: Dennis Wat
Publisher: BoD – Books on Demand
ISBN: 1789841453
Category : Medical
Languages : en
Pages : 138

Book Description
Cystic Fibrosis - Heterogeneity and Personalized Treatment provides the latest research and clinical evidence for clinicians, scientists and researchers involved in the care of patients with cystic fibrosis (CF). This book outlines the burden of the CF microbiome, utilisation of CF registries to impact future care, the sequelae of hepatobiliary complication, the use of upcoming technologies to provide patient-centred care, and provides an overview of cystic fibrosis transmembrane regulator (CFTR) modulators. Looking after patients with CF is highly rewarding, allowing those of us to combine our dedication and problem-solving skills to create a personalized approach. This book is invaluable for those involved in the care of CF patients.

Hodson and Geddes' Cystic Fibrosis

Hodson and Geddes' Cystic Fibrosis PDF Author: Andrew Bush
Publisher: CRC Press
ISBN: 1000988538
Category : Medical
Languages : en
Pages : 747

Book Description
Cystic Fibrosis has seen dramatic advances in treatment since the last edition, including targeted cystic fibrosis transmembrane conductance regulator (CFTR) protein modulators for most CFTR gene abnormalities. This new fifth edition is an update and expansion of the rapid clinical and scientific advances in improving prognosis, and the impact of COVID-19, all of which has transformed conventional models of care. It covers basic science, such as how detailed understanding of the biology of the CFTR gene and protein has led to novel and beneficial therapies, as well as all aspects of clinical management in high-, middle- and low-income settings and the voices of individuals with CF from across the world. It will be a useful reference for clinicians, including all levels of trainees, across the whole multidisciplinary team, scientists and students. Key Features • Follows an appealing organization of chapters, by developing fundamental knowledge of the reader before moving on to more complex or developing topics. • Presents a comprehensive, authoritative and up-to-date text, integrating fundamental science and clinical aspects of cystic fibrosis providing an attractive read for clinicians, trainee doctors and scientists. • Draws on global expertise and reflects best evidence-based practice from experts conducting cutting-edge clinical and basic science research from around the world.

Registries for Evaluating Patient Outcomes

Registries for Evaluating Patient Outcomes PDF Author: Agency for Healthcare Research and Quality/AHRQ
Publisher: Government Printing Office
ISBN: 1587634333
Category : Medical
Languages : en
Pages : 385

Book Description
This User’s Guide is intended to support the design, implementation, analysis, interpretation, and quality evaluation of registries created to increase understanding of patient outcomes. For the purposes of this guide, a patient registry is an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy purposes. A registry database is a file (or files) derived from the registry. Although registries can serve many purposes, this guide focuses on registries created for one or more of the following purposes: to describe the natural history of disease, to determine clinical effectiveness or cost-effectiveness of health care products and services, to measure or monitor safety and harm, and/or to measure quality of care. Registries are classified according to how their populations are defined. For example, product registries include patients who have been exposed to biopharmaceutical products or medical devices. Health services registries consist of patients who have had a common procedure, clinical encounter, or hospitalization. Disease or condition registries are defined by patients having the same diagnosis, such as cystic fibrosis or heart failure. The User’s Guide was created by researchers affiliated with AHRQ’s Effective Health Care Program, particularly those who participated in AHRQ’s DEcIDE (Developing Evidence to Inform Decisions About Effectiveness) program. Chapters were subject to multiple internal and external independent reviews.

Making Medicines Affordable

Making Medicines Affordable PDF Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309468086
Category : Medical
Languages : en
Pages : 235

Book Description
Thanks to remarkable advances in modern health care attributable to science, engineering, and medicine, it is now possible to cure or manage illnesses that were long deemed untreatable. At the same time, however, the United States is facing the vexing challenge of a seemingly uncontrolled rise in the cost of health care. Total medical expenditures are rapidly approaching 20 percent of the gross domestic product and are crowding out other priorities of national importance. The use of increasingly expensive prescription drugs is a significant part of this problem, making the cost of biopharmaceuticals a serious national concern with broad political implications. Especially with the highly visible and very large price increases for prescription drugs that have occurred in recent years, finding a way to make prescription medicinesâ€"and health care at largeâ€"more affordable for everyone has become a socioeconomic imperative. Affordability is a complex function of factors, including not just the prices of the drugs themselves, but also the details of an individual's insurance coverage and the number of medical conditions that an individual or family confronts. Therefore, any solution to the affordability issue will require considering all of these factors together. The current high and increasing costs of prescription drugsâ€"coupled with the broader trends in overall health care costsâ€"is unsustainable to society as a whole. Making Medicines Affordable examines patient access to affordable and effective therapies, with emphasis on drug pricing, inflation in the cost of drugs, and insurance design. This report explores structural and policy factors influencing drug pricing, drug access programs, the emerging role of comparative effectiveness assessments in payment policies, changing finances of medical practice with regard to drug costs and reimbursement, and measures to prevent drug shortages and foster continued innovation in drug development. It makes recommendations for policy actions that could address drug price trends, improve patient access to affordable and effective treatments, and encourage innovations that address significant needs in health care.

Pediatric Respiratory Disease, An Issue of Clinics in Chest Medicine, E-Book

Pediatric Respiratory Disease, An Issue of Clinics in Chest Medicine, E-Book PDF Author: Erick Forno
Publisher: Elsevier Health Sciences
ISBN: 0443130043
Category : Medical
Languages : en
Pages : 273

Book Description
In this issue of Clinics in Chest Medicine, guest editors Drs. Erick Forno and Gregory Sawicki bring their considerable expertise to the topic of Pediatric Respiratory Disease. Top experts discuss COVID-19 in pediatric populations; environmental exposures and pediatric respiratory disease; genetics and genomics in childhood respiratory diseases; microbiome and metabolome: their role in pediatric respiratory disease; and more. - Contains 21 relevant, practice-oriented topics including sickle cell disease and the lung; pediatric lung transplantation; health equity and advocacy in pediatric pulmonology; pediatric pulmonology in low-middle income countries; childhood interstitial lung diseases; primary cilliary dyskinesia; and more. - Provides in-depth clinical reviews on pediatric respiratory disease, offering actionable insights for clinical practice. - Presents the latest information on this timely, focused topic under the leadership of experienced editors in the field. Authors synthesize and distill the latest research and practice guidelines to create clinically significant, topic-based reviews.

Cystic Fibrosis

Cystic Fibrosis PDF Author: Stephanie Duggins Davis
Publisher: Springer Nature
ISBN: 3030423824
Category : Medical
Languages : en
Pages : 525

Book Description
This book provides a comprehensive overview of the multisystem disease, cystic fibrosis, for both pediatric and adult patients. Written by experts in the field, the text outlines the progressive nature of CF as well as the impact of this autosomal recessive disease on the respiratory, gastrointestinal, endocrine, rheumatologic, and renal systems, as well as the patient’s mental health. The book begins with a chapter describing the history of cystic fibrosis and how the face of this life-shortening disease has changed over the past several decades. The following chapters elucidate the pathophysiology of how cystic fibrosis impacts each organ system. Current management and therapeutics are detailed with step-by-step guidelines for clinicians. This book is unique in that it highlights the entire person, not just the respiratory system, with detailed inclusion of the patient perspectives throughout, informing practice standards and considerations. This is an ideal guide for pediatric and adult physicians who care for patients with cystic fibrosis, as well as respiratory therapists, physical therapists, nurses, nutritionists, and pharmacists who care for these patients.

Ion Channels as Targets in Drug Discovery

Ion Channels as Targets in Drug Discovery PDF Author: Gary Stephens
Publisher: Springer Nature
ISBN: 3031521978
Category :
Languages : en
Pages : 540

Book Description