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Latest Rare diseases Stories

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...

2014-03-31 08:29:52

Defitelio®? (defibrotide) Launch Announced During the 40th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) 2014 DUBLIN and VILLA GUARDIA, Italy, March 31, 2014 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and Gentium S.p.A., a Jazz Pharmaceuticals company, today announced the commencement of the European commercial launch of Defitelio®(?) (defibrotide), the first licensed product for the treatment of severe hepatic veno-occlusive...

2014-03-28 11:31:26

Researchers at the UC San Diego School of Medicine have demonstrated for the first time that veterans of the 1990-91 Persian Gulf War who suffer from "Gulf War illness" have impaired function of mitochondria – the energy powerhouses of cells. The findings, published in the March 27, 2014 issue of PLOS ONE, could help lead to new treatments benefitting affected individuals – and to new ways of protecting servicepersons (and civilians) from similar problems in the future, said principal...

2014-03-26 16:25:21

NASHVILLE, Tenn., March 26, 2014 /PRNewswire-USNewswire/ -- Two pediatricians whose research led to the need for early identification of and treatment of severe combined immunodeficiency (SCID) and its inclusion in newborn screening have received the March of Dimes/Col. Harland D. Sanders Lifetime Achievement Award in Genetics. Children affected with SCID are at risk of developing life-threatening infections because they lack a normal immune system. The disorder became familiar to...

2014-03-26 16:18:12

In mice, dietary changes slow down progression of the disease Working with genetically engineered mice, Johns Hopkins neuroscientists report they have identified what they believe is the cause of the vast disintegration of a part of the brain called the corpus striatum in rodents and people with Huntington's disease: loss of the ability to make the amino acid cysteine. They also found that disease progression slowed in mice that were fed a diet rich in cysteine, which is found in foods...


Word of the Day
jument
  • A beast of burden; also, a beast in general.
'Jument' ultimately comes from the Latin 'jugum,' yoke.
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