منابع مشابه
Global effort against rare and orphan diseases.
T he fifth International Congress on Rare Pulmonary Diseases and Orphan Drugs will take place in Milan, Italy, on February 8–9, 2013 (www.pulmonaryrarediseases. com). 10 yrs have passed since the first Congress was held. Since then, the Congress has taken place regularly every 2 yrs, with growing success and ever increasing numbers of participants and speakers from around the world. The Interna...
متن کاملDrugs Against Rare Diseases: Are The Regulatory Standards Higher?
The US Food and Drug Administration (FDA) recently issued a draft Guidance for Industry for Rare Diseases: Common Issues in Drug Development (referred to as "Rare Diseases Guidance"). In our opinion, the FDA should consider: (a) explicitly acknowledging the standards are higher for rare diseases for the reasons presented in this article; and (b) illustrating innovative development pathways that...
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: Journal of Pediatric Gastroenterology & Nutrition
سال: 2010
ISSN: 0277-2116
DOI: 10.1097/mpg.0b013e3181b47b49