Children with rare diseases of neutrophil granulocytes: from therapeutic orphans to pioneers of individualized medicine
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چکیده
منابع مشابه
Patients with rare diseases: from therapeutic orphans to pioneers of personalized treatments
“Nature is nowhere accustomed more openly to display her secret mysteries than in cases where she shows traces of her workings apart from the beaten path; nor is there any better way to advance the proper practice of medicine than to give our minds to the discovery of the usual law of nature, by the careful investigation of cases of rarer forms of disease. For it has been found in almost all th...
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➣ Although definitions vAry by jurisdiction, diseAses that affect approximately 1 in 2000 people are considered rare. 1 Canada is one of only a few developed countries without a national " orphan drug " program to protect patients with rare diseases from exorbitant drug costs. Public debate concerning government funding for these drugs is periodically stimulated by stories of patients who are d...
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ژورنال
عنوان ژورنال: Hematology
سال: 2016
ISSN: 1520-4391,1520-4383
DOI: 10.1182/asheducation-2016.1.33