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Latest Sanfilippo Syndrome Stories

2014-07-23 08:30:28

ArmaGen to Receive up to Approximately $225 Million Including Initial Upfront Payment of $15 Million in Cash and Equity, an Additional Equity Investment, R&D Funding, Development and Sales Milestones, in Addition to Future Royalties CALABASAS, Calif., July 23, 2014 /PRNewswire/ -- ArmaGen, a privately held biotechnology company focused on developing revolutionary therapies to treat severe neurological disorders, announced today that it has entered into a worldwide licensing and...

2014-06-03 08:31:40

LEXINGTON, Mass., June 3, 2014 /PRNewswire/ -- Synageva BioPharma Corp. (Synageva) (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today announced data presentations and a company-sponsored satellite symposium at the European Atherosclerosis Society (EAS) Congress being held May 31-June 3, 2014 in Madrid, Spain. Logo - http://photos.prnewswire.com/prnh/20140522/90728 Sebelipase alfa continues to improve disease-related abnormalities...

2014-06-02 12:26:30

ORLANDO, Fla., June 2, 2014 /PRNewswire/ -- There is a rock anthem titled "School's Out for Summer". While most kids were celebrating their last day of school this week, 12 year-old Luke McMahon decided he would celebrate by singing for a friend in need. Luke's friend is Blair Chapin, also twelve-years-old. Diagnosed with Sanfilippo Syndrome at the age of 6, Blair has slowly been dragged down by a disease whose life expectancy is in the teens. Blair no longer speaks, cannot feed...

2014-05-30 16:23:28

ORLANDO, Fla., May 30, 2014 /PRNewswire/ -- Blair Chapin, a twelve-year-old little girl is dying. Diagnosed with Sanfilippo Syndrome at the age of 6, Blair has slowly been dragged down by a disease whose life expectancy is in the teens. Blair no longer speaks, cannot feed herself and is becoming unstable on her feet. Her father, Roger Chapin of Orlando, has taken to social media with a campaign to "Sing for Blair, Fund a Cure", in an effort to raise awareness and research dollars...

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...

2014-03-31 09:52:21

New insight has been gained into treating an inherited disorder that creates serious neurological and behavioral disabilities in children and usually leads to death in the teen years. In a recent study into the effects of human umbilical cord blood mononuclear cells (hUCB MNCs) when they are injected to counter the symptoms and progression of Sanfilippo syndrome type III B (MPS III B), researchers found that repeated injections into laboratory mice modeled with the disorder had clear...

2014-03-11 04:21:27

BARCELONA, Spain, March 11, 2014 /PRNewswire/ -- - This gene therapy program is being developed in a public-private partnership between ESTEVE and the research team of Dr. Fatima Bosch at the Center for Biotechnology and Gene Therapy of the Universitat Autonoma de Barcelona (UAB) - ESTEVE announce the signing of agreements with REGENX and GENETHON which will allow it to initiate its phase I/II clinical trial in 2015 -...


Word of the Day
mallemaroking
  • Nautical, the visiting and carousing of sailors in the Greenland ships.
This word is apparently from a confusion of two similar Dutch words: 'mallemerok,' a foolish woman, and 'mallemok,' a name for some persons among the crew of a whaling vessel.