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Latest Rare diseases Stories

2013-08-26 08:28:34

Unique Free Event Provides Look at the Complicated Process of Drug Development CAMBRIDGE, Mass., Aug. 26, 2013 /PRNewswire-USNewswire/ -- The ALS Therapy Development Institute (ALS TDI) announced today that its ninth annual Leadership Summit will take place on Thursday, October 3, 2013 at the Westin Copley Place Hotel in Boston, MA. Hosted by the world's first non-profit biotech, this public event explores the latest trends in drug discovery, partnering and financing drug...

2013-08-19 10:35:26

UCSF study shows potential for new treatment strategy UC San Francisco scientists working in the lab used a chemical found in an anti-wrinkle cream to prevent the death of nerve cells damaged by mutations that cause an inherited form of Parkinson’s disease. A similar approach might ward off cell death in the brains of people afflicted with Parkinson’s disease, the team suggested in a study reported online in the journal Cell on August 15. The achievement marks a pharmacologic...

2013-08-13 12:26:43

WASHINGTON, Aug. 13, 2013 /PRNewswire-USNewswire/ -- Today, The ALS Association announced its award of $4.3 million in new research grants supporting 35 new projects. These awards are part of its Translational Research Advancing Therapy (TREAT ALS(TM)) program, through which The Association funds a diverse portfolio of amyotrophic lateral sclerosis (ALS) research to find treatments and a cure for ALS. ALS, which is also known as Lou Gehrig's Disease, is a progressive...

2013-08-12 23:00:28

DailyGossip.org reviews one of the latest kidney disease treatments: the Kidney Disease Solution System. (PRWEB) August 12, 2013 The Kidney Disease Solution review published by http://www.DailyGossip.org on August 12, shows that this efficient treatment has already been used by thousands of people from all over the world, who managed to overcome their kidney disease. The "Kidney Disease Solution System" developed by Duncan Capicchiano provides users a step-by-step plan on how to...

2013-08-12 14:10:30

Researchers have discovered how genetic mutations linked to Parkinson's disease might play a key role in the death of brain cells, potentially paving the way for the development of more effective drug treatments. In the new study, published in Nature Neuroscience, a team of researchers from UCL, the University of Cambridge and the University of Sheffield showed how defects in the Parkinson's gene Fbxo7 cause problems with 'mitaphagy' – an essential process through which our bodies are...

2013-08-07 16:24:09

Be Part of the Cure for "Epidermolysis Bullosa (EB) - The Worst Disease You've Never Heard Of.(TM)" NEW YORK, Aug. 7, 2013 /PRNewswire-USNewswire/ -- The Inaugural Mats Wilander Foundation Tennis Pro-Am benefiting debra of America will be held Wednesday, August 28 from 8:30 A.M. to 2 P.M. at the legendary 100-year-old West Side Tennis Club in Forest Hills, Queens, New York. West Side Tennis Club was the former site of the US Open Tennis Championships for over six decades. The event is open...

2013-08-07 12:27:11

Seeking to Protect Infants and Children from Lifelong Neurological Impairment DANBURY, Conn., Aug. 7, 2013 /PRNewswire-USNewswire/ -- A 14-year-old Connecticut boy has joined the National Organization for Rare Disorders (NORD) in alerting pediatricians and other physicians to a rare medical condition that can leave babies with lifelong disability. (Photo: http://photos.prnewswire.com/prnh/20130807/DC60396) (Logo: http://photos.prnewswire.com/prnh/20110719/DC37656LOGO-b) The boy,...

2013-08-06 23:18:46

University of Rochester Fellow Dr. Jeffrey Statland to Evaluate Electrical Impedance Myography Technology for FSHD Treatment Lexington, Massachusetts (PRWEB) August 06, 2013 Today, the FSH Society, a Massachusetts based non-profit that is a world leader in combating facioscapulohumeral dystrophy (FSHD), announced that it has awarded a $48,909 research grant to Dr. Jeffrey Statland, University of Rochester Fellow and Senior Instructor in the Department of Neurology. The grant will fund Dr....

2013-08-05 23:02:03

Bill Corr to Kick Off Conference 30 Years After Passage of Orphan Drug Act. Horsham, PA (PRWEB) August 05, 2013 Bill Corr, deputy secretary of the U.S. Department of Health and Human Services, will deliver the keynote address at the third annual U.S. Conference on Rare Diseases and Orphan Products in Bethesda, Md., on Oct. 7, 2013. Corr was vital in the passage of the Orphan Drug Act (ODA), serving as counsel to the U.S. House of Representatives’ Subcommittee on Health and the...

2013-08-05 12:27:54

TOWSON, Md., Aug. 5, 2013 /PRNewswire/ -- The Immune Deficiency Foundation (IDF), the national patient organization for persons with primary immunodeficiency diseases, commends the state of Ohio for their decision to add Severe Combined Immune Deficiency Disease (SCID), to the list of conditions that all newborns in the state are screened for at birth beginning July 29, 2013. SCID, sometimes referred to as bubble boy disease, is a primary immunodeficiency disease that is considered...