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Last updated on April 20, 2014 at 21:20 EDT

Latest Rare diseases Stories

2014-04-16 04:21:57

STOCKHOLM, April 16, 2014 /PRNewswire/ -- Wilson Therapeutics, a biopharmaceutical company, today announced that it has closed a $40 million Series B financing co-led by new investors, Abingworth LLP and MVM Life Science Partners LLP ("MVM"). The company's founding investor, HealthCap, also participated in the round. "We are pleased with this significant support from our new as well as existing investors, who represent some of the most experienced in the global life sciences...

2014-04-15 23:02:32

Transparency Market Research Report Added "Amyotrophic Lateral Sclerosis Market - Global Industry Analysis, Size, Share, Growth, Trends And Forecast, 2013 - 2019" to its database. Browse Full Report: http://www.transparencymarketresearch.com/amyotrophic-lateral-sclerosis.html. Albany, New York (PRWEB) April 15, 2014 Amyotrophic lateral sclerosis (ALS) is a neurological disease and also termed as Lou Gehrig’s disease. The disease affects the motor neurons, which control the...

2014-04-15 14:45:09

The International and American Associations for Dental Research (IADR/AADR) have published a paper titled "Rare Bone Diseases and Their Dental, Oral, and Craniofacial Manifestations." The complete review by researchers Sunday O. Akintoye, Andrea B. Burke, Alison M. Boyce, Michael Collins, Brian L. Foster, Rachel I. Gafni, Janice S. Lee, Mary Scott Ramnitz, Martha J. Somerman and J. Timothy Wright is published in the OnlineFirst portion of the IADR/AADR Journal of Dental Research (JDR)....

2014-04-07 08:29:11

STOCKHOLM and CHESTERBROOK, Pa., April 7, 2014 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi) and Auxilium Pharmaceuticals, Inc. (Nasdaq: AUXL) today announced that Sobi became the Market Authorisation Holder (MAH) for Xiapex® (collagenase clostridium histolyticum), in 28 EU member countries, Norway, and Iceland on April 3, 2014. Xiapex/ XIAFLEX® is a first-in-class biologic approved in the US, EU, Canada and Australia for the treatment of adult Dupuytren's contracture...

2014-04-04 23:40:53

Fundraising gala for Tuberous Sclerosis Complex (TSC) will be held at Lake Forest’s Deer Path Inn on May 15th, Global TSC Awareness Day. Lake Forest, IL (PRWEB) April 03, 2014 WISH I MAY, Bcureful’s third annual gala and wine dinner organized to raise funds in support of research and a cure for Tuberous Sclerosis Complex (TSC), a genetic disorder that effects one in 6,000 newborns, is set for Thursday, May 15 at the Deer Path Inn of Lake Forest. The wine dinner presents a menu...

2014-04-04 12:27:44

NORD to Present Awards at "Portraits of Courage" Celebration on May 8th in Washington, DC WASHINGTON, April 4, 2014 /PRNewswire-USNewswire/ -- Rare disease patient advocates, two members of Congress and companies that have brought novel new therapies to patients will be honored at the "Portraits of Courage Celebration" of the National Organization for Rare Disorders (NORD) on Thursday, May 8, at the National Building Museum in Washington, DC....

2014-04-01 23:23:22

Dr. Beverly Davidson Researches Novel Treatments for Neurological Diseases Philadelphia, PA (PRWEB) April 01, 2014 Beverly L. Davidson, Ph.D., a nationally prominent expert in gene therapy, is joining The Children’s Hospital of Philadelphia (CHOP) today. Dr. Davidson, who investigates gene therapy for neurodegenerative diseases, arrives from the Center for Gene Therapy at the University of Iowa. She served as associate director at that Center, as well as director of the Gene Therapy...

2014-04-01 16:25:56

EHE International Donates Window Display to Augie's Quest to Raise Awareness of ALS and Promote Participation in Funding Research for Treatments and a Cure CAMBRIDGE, Mass., April 1, 2014 /PRNewswire/ -- During the month of April, Zumba® Fitness' campaign benefiting Augie's Quest, "Together We Dance(TM) to Cure ALS," will be featured in a window display in Rockefeller Center. Augie's Quest is a fundraising initiative that supports the nonprofit, ALS Therapy Development Institute...

2014-03-31 12:29:17

WASHINGTON, March 31, 2014 /PRNewswire-USNewswire/ -- In work supported by The ALS Association, researchers have discovered a new ALS-causing gene and have linked its function to that of another prominent disease gene. The study was published in the journal Nature Neuroscience. http://photos.prnewswire.com/prnvar/20131211/MM32178LOGO ALS (amyotrophic lateral sclerosis), otherwise known as Lou Gehrig's Disease, is a progressive neurodegenerative disease that affects neurons (nerve...

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...