منابع مشابه
Drugs for exceptionally rare diseases: do they deserve special status for funding?
Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening. Low patient numbers make it difficult for pharmaceutical companies to recoup research and development costs, and consequently these medicines are generally expensive on a per patient basis. European Union (EU) regulations promote the development of orphan drugs; but ...
متن کاملGood ideas deserve repeating.
To the Editor:—In a recent letter, Erickson and Lanier reported on a useful method to preoxygenate claustrophobic patients who will not tolerate a facemask, by having the patients hold the L-connector of the anesthesia circuit in their mouth. They may be interested to know that an identical method, complete with an almost identical photograph, was published as a correspondence by Keifer and Sti...
متن کاملIdentifying rare events in rare diseases.
Utilizing genomic signatures from diagnostic tumor samples to forecast clinical behavior and response to therapy has long been a goal, and we are now poised to further refine how we can identify the relatively rare patients with aggressive neuroblastoma masquerading as patients with a more benign form of the disease. Clin Cancer Res; 21(8); 1782-5. ©2014 AACR. See related article by Oberthuer e...
متن کاملEpigenetics in Rare Diseases
By definition, a rare disease is any condition that affects a small percentage of the population. However, the cutoff number for which a disease is considered as rare varies with different regions. In the United States, the cutoff was fewer than 200,000 people [1] and while in Japan, it was fewer than 50,000 [2]. For Europe, rare diseases are life-threatening or chronically debilitating ones in...
متن کامل[Rare diseases in paediatrics].
Few areas of paediatric care are as susceptible to the revision of lines of action, and require such professional training, as the care of the child with a rare disease (RD) and his/her family, especially if there are malformations. The lifelong impact, which many RDs entail, make continuous monitoring of the child compulsory, centred both on the evolutionary aspects of the RD and on the family...
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ژورنال
عنوان ژورنال: Eurosurveillance
سال: 1999
ISSN: 1560-7917
DOI: 10.2807/esm.04.01.00049-en