Treating Rare and Neglected Pediatric Diseases: Promoting the Development of New Treatments and Cures

Treating Rare and Neglected Pediatric Diseases: Promoting the Development of New Treatments and Cures PDF

Author: Health, Education, Labor, and Pensions United States Senate Committee

Publisher:

Published: 2013-04-28

Total Pages: 134

ISBN-13: 9781484831120

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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.

Treating Rare and Neglected Pediatric Diseases

Treating Rare and Neglected Pediatric Diseases PDF

Author: United States. Congress

Publisher: Createspace Independent Publishing Platform

Published: 2017-12-11

Total Pages: 134

ISBN-13: 9781981610907

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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.

S. Hrg. 111-1147

S. Hrg. 111-1147 PDF

Author: U. S. Government Printing Office (Gpo)

Publisher: BiblioGov

Published: 2013-09

Total Pages: 138

ISBN-13: 9781289375812

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The United States Government Printing Office (GPO) was created in June 1860, and is an agency of the U.S. federal government based in Washington D.C. The office prints documents produced by and for the federal government, including Congress, the Supreme Court, the Executive Office of the President and other executive departments, and independent agencies. A hearing is a meeting of the Senate, House, joint or certain Government committee that is open to the public so that they can listen in on the opinions of the legislation. Hearings can also be held to explore certain topics or a current issue. It typically takes between two months up to two years to be published. This is one of those hearings.

Undiagnosed and Rare Diseases in Children, An Issue of Pediatric Clinics of North America, E-Book

Undiagnosed and Rare Diseases in Children, An Issue of Pediatric Clinics of North America, E-Book PDF

Author: Robert M. Kliegman

Publisher: Elsevier Health Sciences

Published: 2016-12-01

Total Pages: 382

ISBN-13: 0323496725

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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.

Drug Development and Rare Pediatric Diseases

Drug Development and Rare Pediatric Diseases PDF

Author: Frederick Johnston

Publisher: Nova Biomedical Books

Published: 2021-08

Total Pages: 122

ISBN-13: 9781536102284

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Over the past few decades, a number of legislative actions have been taken to encourage the development of therapies to treat people affected by rare diseases and to encourage therapies for pediatric patients. During this same time period, the Food and Drug Administration (FDA) has invested substantial effort in support of these same goals. As described in this book, recent legislative actions have prompted new initiatives. Continuing the response to the laws' specific requirements and FDA's commitment under PDUFA, this book includes input from the public and FDA and discusses the many complex issues involved in medical product development for people with rare diseases. Following a brief overview of both legislative and FDA efforts over the years to foster development of therapies for these patients, the book summarizes the three days of the public meeting and presents FDA's strategic plan for accelerating the development of the urgently needed therapies for pediatric rare diseases.

Rare Diseases and Orphan Products

Rare Diseases and Orphan Products PDF

Author: Institute of Medicine

Publisher: National Academies Press

Published: 2011-04-03

Total Pages: 442

ISBN-13: 0309158060

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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.

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