How really rare are rare disease? The intriguing case of independent compound mutations in the long QT syndrome. J Cardiovasc Electrophysiol 2003;14:1120-1.↵ Schwartz P.J. , et al . How really rare are rare diseases?: The intriguing case of independent compound mutations in the long QT ...
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While Fields condition may become more widespread, what about rare diseases that show little sign of surviving on their own? One such disease is Kuru. Kuru The average person's chances of contracting or developing Kuru are virtually nonexistent. To catch this fatal neurological disease, you'd ...
are often caused by aberrations of a single gene. In rare diseases, we may study how specific genetic defects can trigger a series of events that lead to the expression of a particular disease. Often, the disease process manifested in a certain rare disease is strikingly similar to the diseas...
BackgroundIt is estimated that at present there are over 10 million rare disease patients in China. Recently an increased focus from policy perspective has been placed on rare diseases management. Improved disease definitions and the releases of local and national rare disease lists are some of the...
Twitter Google Share on Facebook rare disease (redirected fromRare diseases) rare disease SeeOrphan disease. McGraw-Hill Concise Dictionary of Modern Medicine. © 2002 by The McGraw-Hill Companies, Inc. Want to thank TFD for its existence?Tell a friend about us, add a link to this page, ...
Rare diseases aren’t just in our name: they are at the core of all that we do. We work side-by-side with rare disease communities to raise awareness, educate, and advocate for improved diagnosis. Together, we can celebrate our successes and strengthen our impact for people living with ra...
SARS-CoV-2 human challenge reveals biomarkers that discriminate early and late phases of respiratory viral infections It’s not always clear whether blood biomarkers are differentially expressed in the time course of viral infections. In this SARS-CoV-2 human challenge study, the authors identify dis...
Rare diseases: companies are looking carefully at the rare disease area now, especially given last year's robust wave of approvals. But there's a steep curve. Beyond small patient populations, firms in this burgeoning space face a host of unique challenges. Noah Pines reports.(MMM & ...
then in the case where there is no other effective treatment for the relevant rare disease, the orphan drug may be granted an exemption of clinical trial and approved for marketing in China, provided that stringent risk control measures are put in place in advance and the MA holder shall perf...