Download or read book Treating Rare and Neglected Pediatric Diseases written by United States. Congress. Senate. Committee on Health, Education, Labor, and Pensions and published by . This book was released on 2012 with total page 144 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Treating Rare and Neglected Pediatric Diseases Promoting the Development of New Treatments and Cures written by Health, Education, Labor, and Pensions United States Senate Committee and published by . This book was released on 2013-04-28 with total page 134 pages. Available in PDF, EPUB and Kindle. Book excerpt: We meet today to discuss a profoundly important issue: the lack of effective treatments for rare and neglected diseases. Over the years, Congress has devoted extraordinary sums for research into major diseases that afflict millions of Americans. But, we've been less generous, and less successful, in mobilizing the research community to come up with therapies and cures for rare and neglected diseases. In the United States, rare diseases are defined as those that affect fewer than 200,000 people. According to the National Institutes of Health, there are nearly 7,000 rare diseases, affecting more than 25 million Americans. Yet, there are FDA-approved treatments for only as few as 200 of these diseases.

Download or read book Treating Rare and Neglected Pediatric Diseases S Hrg 111 1147 July 21 2010 111 2 Hearing written by and published by . This book was released on 2013 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Treating Rare and Neglected Pediatric Diseases written by United States. Congress and published by Createspace Independent Publishing Platform. This book was released on 2017-12-11 with total page 134 pages. Available in PDF, EPUB and Kindle. Book excerpt: Treating rare and neglected pediatric diseases : promoting the development of new treatments and cures : hearing of the Committee on Health, Education, Labor, and Pensions, United States Senate, One Hundred Eleventh Congress, second session ... July 21, 2010.

Download or read book Treating Rare and Neglected Pediatric Diseases Promoting the Development of New Treatments and Cures written by United States. Congress. Senate. Committee on Health, Education, Labor, and Pensions and published by . This book was released on 2012 with total page 130 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Treating Rare and Neglected Pediatric Diseases written by United States. Congress. Senate. Committee on Health, Education, Labor, and Pensions and published by . This book was released on 2012 with total page 130 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Rare Diseases and Orphan Products written by Institute of Medicine and published by National Academies Press. This book was released on 2011-04-03 with total page 442 pages. Available in PDF, EPUB and Kindle. Book excerpt: Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.

Download or read book Undiagnosed and Rare Diseases in Children An Issue of Pediatric Clinics of North America E Book written by Robert M. Kliegman and published by Elsevier Health Sciences. This book was released on 2016-12-01 with total page 382 pages. Available in PDF, EPUB and Kindle. Book excerpt: Dr. Kliegman and Dr. Bordini have written a primer on Undiagnosed and Rare Diseases in Children. Leading experts have presented the current knowledge in the following areas: How Doctor’s Think: Common Diagnostic Errors in Clinical Judgment; Team-Based Approach to Undiagnosed and Rare Diseases; Ending a Diagnostic Odyssey: Family Education Counselling and Their Response to Eventual Diagnosis; Eczema and Urticaria as Manifestations of Undiagnosed and Rare Diseases; Usual and Unusual Manifestations of Familial Hemophagocytic and Langerhans Cell Histiocytosis Syndromes; When Autistic Behavior Suggests a Disease Other than Classic Autism; Non-classic Inflammatory Bowel Disease in Young Infants; IPEX and Other Disorders; Usual and Unusual Presentation of Mitochondrial Disorders; When to Suspect Auto-inflammatory/Recurrent Fever Syndromes; Primary and Secondary Causes of Autonomic Dysfunction; Usual and Unusual Manifestations of Systemic and CNS Vasculitis; Fever of Unknown Origin; Differentiating Familial Neuropathies from Guillain-Barre Syndrome; and Munchausen by Proxy: A Factitious Undiagnosed Disease. Readers will come away with cutting-edge information to use immediately in their clinical management of patients.

Download or read book Rare Diseases written by Marcia Crosse and published by . This book was released on 2016-05-04 with total page 32 pages. Available in PDF, EPUB and Kindle. Book excerpt: Almost 7,999 rare diseases, most of which are serious or life-threatening, affect more than 25 million Americans. About half of all rare diseases affect children, and few of these diseases have viable treatments. To encourage the development of drugs to treat or prevent rare pediatric diseases, the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012 authorized FDA to award a priority review voucher to a drug sponsor upon approval of that sponsor's drug to treat a rare pediatric disease. A drug sponsor can later redeem the voucher when submitting another new drug application to treat any disease or condition in adults or children, or sell or transfer the voucher to another sponsor. A voucher entitles a sponsor to a 6-month priority review by FDA rather than the 10-month standard review. This report examined what is known abut the effectiveness of the pediatric voucher program. Table and figure. This is a print on demand report.

Download or read book Investing to Overcome the Global Impact of Neglected Tropical Diseases written by World Health Organization and published by World Health Organization. This book was released on 2015-08-05 with total page 211 pages. Available in PDF, EPUB and Kindle. Book excerpt: "The presence, or absence, of neglected tropical diseases (NTDs) can be seen as a proxy for poverty and for the success of interventions aimed at reducing poverty. Today, coverage of the public-health interventions recommended by the World Health Organization (WHO) against NTDs may be interpreted as a proxy for universal health coverage and shared prosperity - in short, a proxy for coverage against neglect. As the world's focus shifts from development to sustainable development, from poverty eradication to shared prosperity, and from disease-specific goals to universal health coverage, control of NTDs will assume an important role towards the target of achieving universal health coverage, including individual financial risk protection. Success in overcoming NTDs is a "litmus test" for universal health coverage against NTDs in endemic countries. The first WHO report on NTDs (2010) set the scene by presenting the evidence for how these interventions had produced results. The second report (2013) assessed the progress made in deploying them and detailed the obstacles to their implementation. This third report analyses for the first time the investments needed to achieve the scale up of implementation required to achieve the targets of the WHO Roadmap on NTDs and universal coverage against NTDs. INVESTING TO OVERCOME THE GLOBAL IMPACT OF NEGLECTED TROPICAL DISEASES presents an investment strategy for NTDs and analyses the specific investment case for prevention, control, elimination and eradication of 12 of the 17 NTDs. Such an analysis is justified following the adoption by the Sixty-sixth World Health Assembly in 2013 of resolution WHA6612 on neglected tropical diseases, which called for sufficient and predictable funding to achieve the Roadmap's targets and sustain control efforts. The report cautions, however, that it is wise investment and not investment alone that will yield success. The report registers progress and challenges and signals those that lie ahead. Climate change is expected to increase the spread of several vector-borne NTDs, notably dengue, transmission of which is directly influenced by temperature, rainfall, relative humidity and climate variability primarily through their effects on the vector. Investments in vector-borne diseases will avoid the potentially catastrophic expenditures associated with their control. The presence of NTDs will thereby signal an early warning system for climate-sensitive diseases. The ultimate goal is to deliver enhanced and equitable interventions to the most marginalized populations in the context of a changing public-health and investment landscape to ensure that all peoples affected by NTDs have an opportunity to lead healthier and wealthier lives."--Publisher's description.

Download or read book Addressing the Neglected Diseases Treatment Gap written by United States. Congress. House. Committee on Foreign Affairs. Subcommittee on Africa, Global Health, Global Human Rights, and International Organizations and published by . This book was released on 2013 with total page 108 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Medical Child Abuse written by Thomas A. Roesler and published by . This book was released on 2009 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Thomas A. Roesler, MD, FAAP and Carole Jenny, MD, MBA, FAAP make the case that the term Munchausen syndrome by proxy should be retired permanently and replaced with a commonsense appreciation that children can be abused by their parents in the medical environment. Physicians who find themselves providing unnecessary and harmful medical care can see the abuse for what it is, another way parents can harm children. the book offers the first detailed and comprehensive description of treatment for this form of child maltreatment.

Download or read book Inequalities in Health written by Nir Eyal and published by Oxford University Press, USA. This book was released on 2013-10 with total page 348 pages. Available in PDF, EPUB and Kindle. Book excerpt: Which inequalities in longevity and health among individuals, groups, and nations are unfair? And what priority should health policy attach to narrowing them? These essays by philosophers, economists, epidemiologists, and physicians attempt to determine how health inequalities should be conceptualized, measured, ranked, and evaluated.

Download or read book Legislative Calendar written by United States. Congress. Senate. Committee on Health, Education, Labor, and Pensions and published by . This book was released on with total page 172 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Safe and Effective Medicines for Children written by Institute of Medicine and published by National Academies Press. This book was released on 2012-10-13 with total page 432 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were designed to encourage more pediatric studies of drugs used for children. The FDA asked the IOM to review aspects of pediatric studies and changes in product labeling that resulted from BPCA and PREA and their predecessor policies, as well as assess the incentives for pediatric studies of biologics and the extent to which biologics have been studied in children. The IOM committee concludes that these policies have helped provide clinicians who care for children with better information about the efficacy, safety, and appropriate prescribing of drugs. The IOM suggests that more can be done to increase knowledge about drugs used by children and thereby improve the clinical care, health, and well-being of the nation's children.

Download or read book Orphan Drugs and Rare Diseases written by David C Pryde and published by Royal Society of Chemistry. This book was released on 2014-07-30 with total page 350 pages. Available in PDF, EPUB and Kindle. Book excerpt: Orphan drugs are designated drug substances that are intended to treat rare or ‘orphan’ diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world’s population; however, individually, any single, rare disease may only affect a handful of people making them commercially unattractive for the biopharmaceutical industry to target. Ground breaking legislation, starting with the Orphan Drug Act that was passed in the US in 1983 to provide financial incentives for companies to develop orphan drugs, has sparked ever increasing interest from biopharmaceutical companies to tackle rare diseases. These developments have made rare diseases, and the orphan drugs that treat them, sufficiently attractive to pharmaceutical development and many pharmaceutical companies now have research units dedicated to this area of research. It is therefore timely to review the area of orphan drugs and some of the basic science, drug discovery and regulatory factors that underpin this important, and growing, area of biomedical research. Written by a combination of academic and industry experts working in the field, this text brings together expert authors in the regulatory, drug development, genetics, biochemistry, patient advocacy group, medicinal chemistry and commercial domains to create a unique and timely reference for all biomedical researchers interested in finding out more about orphan drugs and the rare diseases they treat. Providing an up-to-date monograph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs and will appeal to medicinal and pharmaceutical chemists, biochemists and anyone working within the fields of rare disease research and drug development or pharmaceuticals in industry or academia.

Download or read book Children in Medical Research written by Lainie Friedman Ross and published by OUP Oxford. This book was released on 2006-02-09 with total page 300 pages. Available in PDF, EPUB and Kindle. Book excerpt: Lainie Ross presents a rigorous critical investigation of the development of policy governing the involvement of children in medical research. She examines the shift in focus from protection of medical research subjects, enshrined in post-World War II legislation, to the current era in which access is assuming greater precedence. Infamous studies such as Willowbrook (where mentally retarded children were infected with hepatitis) are evidence that before the policy shift protection was not always adequate, even for the most vulnerable groups. Additional safeguards for children were first implemented in many countries in the 1970s and 1980s; more recent policies and guidelines are trying to promote greater participation. Ross considers whether the safeguards work, whether they are fair, and how they apply in actual research practice. She goes on to offer specific recommendations to modify current policies and guidelines. Ross examines the regulatory structures (e.g. federal regulations and institutional review boards), the ad hoc policies (e.g. payment in pediatric research and the role of schools as research venues), the actual practices of researchers (e.g. the race/ethnicity of enrolled research subjects or the decision to enroll newborns) as well as the decision-making process (both parental permission and the child's assent), in order to provide a broad critique. Some of her recommendations will break down current barriers to the enrolment of children (e.g. permitting the payment of child research subjects; allowing healthy children to be exposed to research that entails more than minimal risk without requiring recourse to 407 panels); whereas other recommendations may create new restrictions (e.g., the need for greater protection for research performed in schools; restrictions on what research should be done in the newborn nursery). The goal is to ensure that medical research is done in a way that promotes the health of current and future children without threatening, to use the words of Hans Jonas, 'the erosion of those moral values whose loss . . . would make its most dazzling triumphs not worth having'.