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Latest Rare diseases Stories

2014-05-12 12:30:47

$100,000 prize and access to additional expertise awarded to support "big data" genetic analysis to provide new clues to the higher incidence of ALS in veterans BRIDGEWATER, N.J., May 12, 2014 /PRNewswire/ -- Sanofi US announced the winner of its second Innovation Challenge: Collaborate Innovate. The winner, Veterans Advancing ALS/MND, is a partnership that comprises Innovative Solutions Consortium and Johns Hopkins University. In the spirit of collaboration, Sanofi's Partners in Patient...

2014-05-12 08:32:50

CLEVELAND, May 12, 2014 /PRNewswire-USNewswire/ -- Abeona Therapeutics, a start-up company created around intellectual property licensed from Nationwide Children's Hospital to develop treatments for Sanfilippo Syndrome Types A and B, has been granted Orphan Drug Designations for its lead investigational therapies by the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development. Following the successful close of seed financing in late 2013, Abeona is raising funds...

2014-05-12 08:31:08

DUBLIN and SAN DIEGO, May 12, 2014 /PRNewswire/ -- - Acquisition of Lumena Pharmaceuticals, a biopharmaceutical company with late stage rare disease pipeline assets - Adds to Shire's rare diseases portfolio and leverages this expertise, and is a perfect combination with Shire's already strong Gastrointestinal (GI) presence - Adds LUM001 in Phase 2 development for four rare and devastating hepatic diseases, two...

2014-05-08 12:29:19

Papa John's Founder, Chairman and CEO John Schnatter Agrees to Serve as National Walk Ambassador WASHINGTON, May 8, 2014 /PRNewswire-USNewswire/ -- Papa John's International, based in Louisville, Kentucky, is helping The ALS Association raise awareness of amyotrophic lateral sclerosis (ALS) and boost participation the Walk to Defeat ALS®, which brings hope to those living with ALS and is an opportunity for individuals with the disease and their families and friends to spend a day...

2014-05-07 23:01:17

With expanded screening test, 50 percent of Ashkenazi Jewish individuals are expected to be identified as carriers of at least one of the 38 diseases New York, NY (PRWEB) May 07, 2014 The Icahn School of Medicine at Mount Sinai today announced the launch of its Expanded Carrier Screening Panel for people of Ashkenazi Jewish descent. This test increases the number of diseases covered from 20 to 38, giving Ashkenazi Jewish individuals a 1 in 2 chance of being a carrier for at least one of...

2014-05-02 12:27:25

WASHINGTON, May 2, 2014 /PRNewswire-USNewswire/ -- William A. Thoet, Chairman of the Board of Trustees of The ALS Association, announced today that Barbara Newhouse has been named President and CEO of The Association after an extensive nationwide search. She will begin work on June 2, 2014. http://photos.prnewswire.com/prnvar/20140502/83774 ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the...

2014-05-01 08:35:15

DUBLIN, May 1, 2014 /PRNewswire/ -- Shire (LSE: SHP, NASDAQ: SHPG) announces unaudited results for the three months to March 31, 2014. Financial Highlights Q1 2014 Growth(1) Product sales $1,308 million +19% Total revenues $1,347 million +18% Non GAAP operating income $591 million +40%...

2014-04-30 23:11:37

The Multiple System Atrophy Coalition announces the debut of "My Hero - An MSA Angel Story," an inspiring video of courage and love at the Brain Health Fair during the annual American Academy of Neurology in Philadelphia, PA. Sarasota, Florida (PRWEB) April 30, 2014 Sarasota resident Alix Sun recently received a call from the Neuro Film Festival that she had been anticipating for several weeks. The call officially confirmed that her entered video “My Hero - an MSA Angel...

2014-04-30 08:34:06

Parion plans to initiate Phase 2 studies of P-1037 for the treatment of cystic fibrosis in the second half of 2014 DURHAM, N.C., April 30, 2014 /PRNewswire/ -- Parion Sciences, a company dedicated to the development of novel treatments for pulmonary and ocular diseases, announced today that it has reached an agreement with Gilead Sciences, Inc. to reacquire the rights to epithelial sodium channel (ENaC) inhibitors identified and developed under a research collaboration announced...

2014-04-29 23:14:57

The 5p- Society is joining with families around the globe that have been affected by Cri Du Chat, to send a message to the world. That message is that these individuals deserve to be recognized for what they can do versus what they cannot do. Lakewood, CA (PRWEB) April 29, 2014 May 4-10, 2014 is Cri Du Chat Syndrome Awareness Week May 5 is officially designated as Cri Du Chat Day in the United States. The 5p- Society of North America, along with support organizations from Argentina,...


Word of the Day
holluschickie
  • A 'bachelor seal'; a young male seal which is prevented from mating by its herd's older males (mated bulls defending their territory).
This comes from the Russian word for 'bachelors.'
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