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Latest Rare diseases Stories

2014-08-16 12:21:14

WASHINGTON, Aug. 16, 2014 /PRNewswire-USNewswire/ -- Today, The ALS Association announced it has surpassed $10 million in "Ice Bucket" donations. Specifically, as of Saturday, August 16, 2014, The ALS Association has received $11.4 million in donations compared to $1.7 million during the same time period last year (July 29 to August 16). These donations have come from existing donors and 220,255 new donors to The Association. http://photos.prnewswire.com/prnvar/20131211/MM32178LOGO...

2014-08-15 20:21:50

CHICAGO, Aug. 15, 2014 /PRNewswire/ -- Having a bucket of ice water dumped on your head may not seem dignified, but it is certainly helping MDA and other ALS-fighting organizations raise awareness and funds as the viral social media craze continues to sweep across America. Today, Steven M. Derks, President and CEO of the Muscular Dystrophy Association, joined the ranks of tens of thousands of people willing to get chilled in the ALS Ice Bucket Challenge. Derks accepted the...

rna targeting of ALS
2014-08-15 09:21:54

Cell Press By targeting RNA molecules that tangle and clump in the nervous systems of patients with the most common genetic form of amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and frontotemporal dementia (FTD), researchers have shown they can effectively limit those damaging elements in cells taken from patients. The results reported in the Cell Press journal Neuron on August 14th show that RNA is a viable drug target for the two overlapping and incurable...

2014-08-13 12:29:02

New White Paper also Available on Disease that is a Leading Cause of Young-Onset Dementia RADNOR, Pa., Aug. 13, 2014 /PRNewswire-USNewswire/ -- Frontotemporal degeneration (FTD), a disease process that results in progressive damage to the temporal and/or frontal lobes of the brain, has been formally included in national research priorities to cure Alzheimer's disease and other dementias by 2025, the Association of Frontotemporal Degeneration (AFTD) announced today....

2014-08-13 12:28:49

Donations Exceed Expectations with $5.7 Million since July 29 WASHINGTON, Aug. 13, 2014 /PRNewswire-USNewswire/ -- In the last two weeks, the Ice Bucket Challenge has quite literally "soaked" the nation. Everyone from Ethel Kennedy to Justin Timberlake has poured a bucket of ice water over her or his head and has challenged others do the same or to make a donation to fight ALS within twenty-four hours. http://photos.prnewswire.com/prnvar/20131211/MM32178LOGO Between July 29 and...

2014-08-13 08:29:24

WASHINGTON, Aug. 13, 2014 /PRNewswire/ -- The National Organization for Rare Disorders (NORD) today announced the agenda for the Rare Diseases and Orphan Product Breakthrough Summit, to be held October 21-22, 2014 at the Hilton Mark Center in Alexandria, VA. Photo - http://photos.prnewswire.com/prnh/20140812/135518 This annual event is the innovative hub for all stakeholders in the rare disease and orphan products community - providing an educational platform for pharmaceutical, biotech,...

2014-08-11 20:24:21

LONDON, Aug. 11, 2014 /PRNewswire/ -- Reportbuyer.com has added a new market research report:Lysosomal Storage Disorder-Pipeline Insights, 2014https://www.reportbuyer.com/product/2278642/-Lysosomal-Storage-Disorder-Pipeline-Insights-2014.htmlSUMMARYDelveInsight's," Lysosomal Storage Disorder-Pipeline Insights, 2014", report provides comprehensive insights about pipeline drugs across this indication. A key objective of the report is to establish the understanding for all the pipeline...

2014-08-11 16:27:52

LYNBROOK, N.Y., Aug. 11, 2014 /PRNewswire/ -- BioSpecifics Technologies Corp. (NASDAQ: BSTC), a biopharmaceutical company developing first in class collagenase-based products marketed as XIAFLEX(®) (collagenase clostridium histolyticum or CCH) in the U.S. and XIAPEX(®) in the EU, today announced its financial results for the second quarter ended June 30, 2014 and provided a corporate update. "This was a momentous quarter of milestones for XIAFLEX both commercially and clinically. We saw...

2014-08-11 12:28:11

WASHINGTON, Aug. 11, 2014 /PRNewswire-USNewswire/ -- The ALS Association announced today that it has awarded 21 new research grants totaling nearly $3.5 million to find treatments and a cure for amyotrophic lateral sclerosis (ALS). The grants were awarded to scientists from laboratories located in nine different states in the United States as well as from the United Kingdom, France, Canada, Switzerland, Israel and Australia. http://photos.prnewswire.com/prnvar/20131211/MM32178LOGO...

2014-08-09 23:04:05

Emily Gimble's new single, “Mighty Like a Rose,” is available for download through many online music distributors. Austin, Texas (PRWEB) August 09, 2014 Pianist, vocalist and newest member of Asleep at the Wheel, Emily Gimble has a brand new single, “Mighty Like a Rose,” currently available for downloads on the web at CD Baby.com, iTunes, Amazon and other online distributors. All proceeds benefit Beyond Batten Disease Foundation (BBDF), a nonprofit organization established to...


Word of the Day
cock-a-hoop
  • Exultant; jubilant; triumphant; on the high horse.
  • Tipsy; slightly intoxicated.
This word may come from the phrase 'to set cock on hoop,' or 'to drink festively.' Its origin otherwise is unclear. A theory, according to the Word Detective, is that it's a 'transliteration of the French phrase 'coq a huppe,' meaning a rooster displaying its crest ('huppe') in a pose of proud defiance.' Therefore, 'cock-a-hoop' would 'liken a drunken man to a boastful and aggressive rooster.'
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