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Medical Clinics of North America, 2019
There are hundreds of rare orphan lung diseases. We have highlighted five of them, one from each of the five major categories of pulmonary disorders: pleuroparenchymal fibroelastosis (a rare diffuse parenchymal lung disease), pulmonary alveolar proteinosis (a rare autoimmune and diffuse parenchymal lung disease), lymphangioleiomyomatosis (a rare cystic
Muhammad Sajawal, Ali +2 more
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There are hundreds of rare orphan lung diseases. We have highlighted five of them, one from each of the five major categories of pulmonary disorders: pleuroparenchymal fibroelastosis (a rare diffuse parenchymal lung disease), pulmonary alveolar proteinosis (a rare autoimmune and diffuse parenchymal lung disease), lymphangioleiomyomatosis (a rare cystic
Muhammad Sajawal, Ali +2 more
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Current Opinion in Anaesthesiology, 2015
Orphan diseases certainly have a challenging impact on anesthesia practice. Low prevalence by definition leads to a profound lack of evidence-based medical knowledge, and anesthetists usually cannot rely on personal experience for handling this unique group of patients.
Johannes, Prottengeier +2 more
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Orphan diseases certainly have a challenging impact on anesthesia practice. Low prevalence by definition leads to a profound lack of evidence-based medical knowledge, and anesthetists usually cannot rely on personal experience for handling this unique group of patients.
Johannes, Prottengeier +2 more
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2016
A 46-year-old Caucasian male was referred to the gastroenterology clinic with abdominal distension and jaundice. His medical history included chronic obstructive pulmonary disease. He had a five pack-year smoking history and was a teetotaller. He had three brothers who died of liver failure aged in their 50s.
Reenam Khan, Phil Newsome
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A 46-year-old Caucasian male was referred to the gastroenterology clinic with abdominal distension and jaundice. His medical history included chronic obstructive pulmonary disease. He had a five pack-year smoking history and was a teetotaller. He had three brothers who died of liver failure aged in their 50s.
Reenam Khan, Phil Newsome
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The Challenges of Orphan Drugs and Orphan Diseases: Real and Imagined
Clinical Pharmacology & Therapeutics, 2012The Orphan Drug Act of 1983 in the United States and similar legislation in Europe in 1999 provided incentives for companies to develop and sell medicines for diseases with a small market. In this Commentary, we outline the European position on the regulation of orphan drugs and explain where it differs from the regulation in the United States.
I, Hudson, A, Breckenridge
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Changing the Course of an Orphan Disease
New England Journal of Medicine, 2021Patients with von Hippel–Lindau (VHL) disease, a rare autosomal dominant inherited disease,1 have a shorter life expectancy than the general population.2,3 A prospective analysis involving 128 pati...
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Orphan drugs—or orphan diseases?
1988In the beginning, the question was that of how to provide drugs for rare diseases; and the beginning in this country was perhaps around 1972 when the ‘Pharmaceutical Working Party’ of the National Economic Development Office discussed the matter and agreed to keep it under review [1].
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Orphan Diseases and Orphan Drugs : Gaucher disease
2014Gaucher disease is an inherited autosomal recessive metabolic defect due to a deficiency of the lysosomal enzyme ß-glucocerebrosidase. The most common is type 1, which can produce a broad range of presentations characterized by cytopenia and involvement of the spleen, liver and bone marrow. Types 2 and 3 are rarest variants that manifest in infancy and
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Recenti progressi in medicina, 1994
Basic investigation in industrial laboratories are mainly addressed to widespread diseases. It follows that: 1) basic research is addressed chiefly to the well known and largely diffuse diseases; 2) rare diseases are not usually studied and therefore little chance is given to their physiopathological or therapeutic knowledge.
A, Loizzo, M T, Tebano
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Basic investigation in industrial laboratories are mainly addressed to widespread diseases. It follows that: 1) basic research is addressed chiefly to the well known and largely diffuse diseases; 2) rare diseases are not usually studied and therefore little chance is given to their physiopathological or therapeutic knowledge.
A, Loizzo, M T, Tebano
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[Orphan drugs and orphan diseases].
Revista de neurologia, 2002To define the concepts of orphan drugs and diseases, the current situation of clinical investigation of them, the attitude of the pharmaceutical industry and aspects of health legislation, especially in the European Community.We review the history of orphan drugs, the name used for those drugs, apparatus, biological agents and dietetic preparations ...
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