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Last updated on April 24, 2014 at 21:24 EDT

Latest Rare diseases Stories

2013-09-24 08:30:52

'One of the most promising private biotechnology companies in the industry' DURHAM, N.C., Sept. 24, 2013 /PRNewswire/ -- Scioderm, Inc., a biopharmaceutical company focused on the development of innovative therapies for treating rare diseases with high unmet need, has been selected by FierceBiotech as one of their "Fierce 15" for 2013. The "Fierce 15" is FierceBiotech's annual list of the top 15 emerging companies in the biotech industry, thus designating Scioderm, Inc. as one of the...

2013-09-23 08:25:03

Two Peer-Reviewed Papers Featured in October Issue of Pediatric Neurology SILVER SPRING, Md., Sept. 23, 2013 /PRNewswire-USNewswire/ -- Today, the Tuberous Sclerosis Alliance (TS Alliance) formally announced newly updated clinical consensus guidelines for the diagnosis, surveillance and management of tuberous sclerosis complex (TSC). To communicate the recommendations to healthcare professionals, the October issue of Pediatric Neurology includes two peer-reviewed papers detailing the...

2013-09-19 16:26:25

LYNBROOK, N.Y., Sept. 19, 2013 /PRNewswire/ -- BioSpecifics Technologies Corp. (NASDAQ: BSTC), a biopharmaceutical company developing first-in-class collagenase-based products marketed as XIAFLEX(®) in the U.S and XIAPEX(®) in the EU, today announced that BioSpecifics' President, Tom Wegman, will present a corporate overview at the Aegis Capital 2013 Healthcare Conference. The presentation will take place on Thursday, September 26, 2013 at 4:00 p.m. EDT (1:00 p.m. PDT) at the...

2013-09-19 10:16:34

A genetic mutation, known as GBA, that leads to early onset of Parkinson's disease and severe cognitive impairment (in about 4 to 7 percent of all patients with the disease) also alters how specific lipids, ceramides and glucosylceramides are metabolized. Mayo Clinic researchers have found that Parkinson's patients who do not carry the genetic mutation also have higher levels of these lipids in the blood. Further, those who had Parkinson's and high blood levels were also more likely to have...

2013-09-18 08:29:01

How Will the Affordable Care Act, Globalization and Current Market Trends Affect Development of Therapies for People with Rare Diseases? WASHINGTON, Sept. 18, 2013 /PRNewswire-USNewswire/ -- One of the hottest trends in the pharmaceutical and biotechnology industries in recent years has been the development of orphan products or treatments for people with rare diseases. (Logo: http://photos.prnewswire.com/prnh/20110719/DC37656LOGO-b) Now, industry-watchers are wondering how legislation such...

2013-09-16 23:18:19

Fourth Annual Celebrity Walk n’ Roll to Raise Funds for FSH Muscular Dystrophy Research: Sunday, October 6 at 10:00 AM in Heritage Park, Irvine, Open to the Public IRVINE, Calif. (PRWEB) September 16, 2013 The FSH Society announced that the Fourth Annual Celebrity Walk n’ Roll fundraiser, hosted by Max Adler of “Glee,” will take place on Sunday, October 6, 2013 at Heritage Park in Irvine from 10:00 a.m. to 2:00 p.m. More than 20 celebrities and athletes will participate in the...

2013-09-16 08:29:11

The multi-state public health initiative offers an affordable, easy-to-use test, accessible from home ATLANTA, Sept. 16, 2013 /PRNewswire/ -- Fighting hereditary disease among Jews is the aim of a multi-state public health initiative launched today, called JScreen. The JScreen program (www.jscreen.org), managed by Emory University School of Medicine's Department of Human Genetics, provides at-home genetic screening and private counseling for people with Jewish lineage to determine...

2013-09-13 23:01:19

Register Now to Attend Premier Conference on Orphan Product Development, Emerging Markets and Outlook for Investment. Horsham, PA (PRWEB) September 13, 2013 The early registration deadline for the third annual U.S. Conference on Rare Diseases and Orphan Products is Monday, Sept. 16. Co-sponsored by the National Organization for Rare Disorders (NORD) and DIA in collaboration with the Food and Drug Administration (FDA) and EURORDIS (Rare Diseases Europe), the conference will take place from...

2013-09-12 23:31:20

New York medical malpractice lawyer David Perecman discusses reasonable precautions and medical malpractice following reports of 13 hospital patients possibly exposed to degenerative Creutzfeldt-Jakob brain disease. New York, New York (PRWEB) September 12, 2013 Thirteen people in Massachusetts and New Hampshire may have been exposed to the rare and fatal brain condition called Creutzfeldt-Jakob Disease (CJD) through potentially contaminated hospital equipment, said CNN(9.5.13).  The...

2013-09-10 08:31:29

Collaboration to Explore Compound that Regulates Neuroinflammation and Apoptosis CAMBRIDGE, Mass., Sept. 10, 2013 /PRNewswire-USNewswire/ -- The ALS Therapy Development Institute (ALS TDI) announced today that it has entered into a research partnership with Anida Pharma, Inc., to investigate a potential treatment for ALS, also known as Lou Gehrig's disease. (Logo: http://photos.prnewswire.com/prnh/20110204/DC42625LOGO) Under the terms of the agreement with Anida, ALS TDI will...