Rare diseases in Romania – a response to ‘transposition and implementation of EU rare diseases policy in Eastern Europe’
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چکیده
منابع مشابه
The present situation of Rare Diseases in Central/Eastern Europe? The role of patient organisations
Results 1, At the level of medical and social services: It is still “incidental” to get to the appropriate expert or centre for diagnosis or treatment. It is difficult to find even the services, because of the lack of suitable “pathways” and referral. There are long delays in obtaining the first appointment, resulting in defencelessness and regional irregularity. The overall consequence is the ...
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CMAJ • NOVEMBER 23, 2010 • 182(17) © 2010 Canadian Medical Association or its licensors E787 The small market for drugs used to treat rare diseases often makes the drugs extremely expensive. Consequently, many pharmaceutical companies may stop manufacturing these drugs or may not initiate research and development into new therapies. Moving these drugs from bench to bedside requires a partnershi...
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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...
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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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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...
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ژورنال
عنوان ژورنال: Expert Review of Pharmacoeconomics & Outcomes Research
سال: 2018
ISSN: 1473-7167,1744-8379
DOI: 10.1080/14737167.2018.1467864