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Rare and Ultra Rare Diseases [PDF]

open access: possibleJournal of Developing Drugs, 2012
In the last years, rare and ultra rare diseases have attracted worldwide more and more attention. However, there is no universal definition of a rare disease: In Japan the term “orphan disease” describes a condition with a prevalence of 2.5 cases per 10,000 population; in the US 7 cases per 10,000 population.
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Rare liver diseases are not rare in China

Liver International, 2022
AbstractRare diseases, also known as orphan diseases, are a group of diseases that affect a relatively small number of people. Low incidence rates, atypical symptoms, imperfect diagnosis criteria and few treatment options impose medical, psychological and financial stress on the local healthcare system.
Tingting Lv, Jidong Jia
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Rare Autoimmune Diseases

2010
Under the term "autoimmune diseases" are comprised a large number of disorders with variable clinical expression which have in common an autoimmune pathogenesis as defined by direct, indirect or circumstantial evidence. Autoimmune diseases may affect a single organ or may determine a multisystem involvement, and most of them cause significant and ...
Arrigo Schieppati, Erica Daina
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[Rare lung diseases].

Recenti progressi in medicina, 2014
"Rare" diseases are a family of different disorders or conditions affecting fewer than 1/2000 in the general population. Among them, 150 involve respiratory system. To date, most reviews in literature focuses on the more important pulmonary lung diseases without taking in account the overall framework and diagnostic and therapeutic algorithms that ...
Franco Salvati   +2 more
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The burden of rare diseases

American Journal of Medical Genetics Part A, 2019
The subject of rare disease numbers is rife with misconceptions, not just in websites and other layman's literature, but also in the medical literature. Various websites mention numbers that are not validated by any solid data, while in turn the medical literature cites the aforementioned websites as sources, thus perpetuating a number of myths about ...
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Undiagnosed and Rare Diseases

Clinics in Perinatology, 2017
Brett J. Bordini, Robert M. Kliegman
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RARE COMPLICATION OF A RARE DISEASE

Chest, 2019
Ali Bin Sarwar Zubairi   +3 more
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Drugs for rare diseases

Nursing, 2020
Kavitha S Dalal, Mary Barna Bridgeman
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