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Last updated on April 21, 2014 at 9:29 EDT

Latest Rare diseases Stories

2013-12-16 08:27:14

NEW YORK, Dec. 16, 2013 /PRNewswire/ -- The newborn screening test for Severe Combined Immune Deficiency (SCID) using the TREC Assay, is 100 percent sensitive and not a single newborn with this life threatening defect has been lost since the test's implementation five years ago. Nevertheless, public health officials still deliberate over the test's economic efficiency and that has resulted in the Jeffrey Modell Foundation's (JMF) newly created economic model, "Decision Tree."...

2013-12-11 12:12:42

A range of studies highlighting late-breaking research advances in the understanding and treatment of blood cancers and bleeding disorders are being presented today during the 55th American Society of Hematology Annual Meeting and Exposition in New Orleans. State-of-the-art genetic sequencing and analytics tools are being employed in medical research to gain an unprecedented understanding of disease mechanisms and treatments. In the search to improve understanding of disease, two studies...

2013-12-11 08:30:31

Copayment Assistance Now Available to Peyronie's Patients GAITHERSBURG, Md., Dec. 11, 2013 /PRNewswire/ -- The HealthWell Foundation, an independent non-profit providing nationwide financial assistance to insured Americans with high out-of-pocket medication expenses, today announced the launch of a new fund to assist patients with Peyronie's disease, the development of fibrous scar tissue inside the penis. The fund provides copayment assistance to people living with Peyronie's who...

2013-12-10 10:29:34

A team led by a longtime Oregon Health & Science University researcher has demonstrated in mice what could be a revolutionary new technique to cure a wide range of human diseases — from cystic fibrosis to cataracts to Alzheimer's disease — that are caused by "misfolded" protein molecules. Misfolded protein molecules, caused by gene mutation, are capable of maintaining their function but are misrouted within the cell and can’t work normally, thus causing disease. The OHSU team...

2013-12-06 08:24:19

Collaboration to Explore Recombinant Human-Derived Monoclonal Antibodies Targeting Misfolded SOD1 CAMBRIDGE, Mass., Dec. 6, 2013 /PRNewswire-USNewswire/ -- The ALS Therapy Development Institute (ALS TDI), together with its wholly-owned subsidiary, Anelixis Therapeutics, announced today that it has formed a research partnership with Neurimmune to advance potential treatments for ALS, also known as Lou Gehrig's disease. The research partnership will explore a human monoclonal antibody...

Study Explains Why Promising Dementia Drugs Failed In Clinical Trials
2013-12-05 19:34:38

Cell Press Alzheimer's disease is the most common cause of dementia among older people, yet there currently are no effective drugs to stop, slow or prevent disease progression. A study online December 5th in the ISSCR's journal Stem Cell Reports, published by Cell Press, provide interesting clues on why non-steroidal anti-inflammatory drugs (NSAIDs), which have successfully treated molecular signs of Alzheimer's disease in cell and animal models, eventually failed in clinical studies....

2013-12-05 08:28:38

LONDON, Dec. 5, 2013 /PRNewswire/ -- A study just published by the Office of Health Economics (OHE) and its collaborators demonstrates how medicines for rare disease may be effectively valued using an innovative approach -- multi-criteria decision analysis (MCDA) -- that can include all relevant stakeholders, including patients' groups. (Logo: http://photos.prnewswire.com/prnh/20130904/PH74260LOGO) Assessing value for treatments of rare diseases has long been a challenge. The small...

2013-12-03 23:02:00

St. Jude Children’s Research Hospital scientists identify enzyme that could lead to better diagnosis and treatment of Alzheimer’s disease and related disorders. Memphis, Tenn. (PRWEB) December 03, 2013 St. Jude Children’s Research Hospital scientists have identified an enzyme that can halt or possibly even reverse the build-up of toxic protein fragments known as plaques in the brains of mice with Alzheimer’s disease. The research appeared in a recent edition of the scientific...

2013-12-02 08:30:54

DURHAM, N.C., Dec. 2, 2013 /PRNewswire/ -- Scioderm, Inc. announced the appointment of John Crowley and William Aliski to the company's Board of Directors. As Chairman & CEO of NASDAQ listed Amicus Therapeutics, John brings a significant rare and orphan disease experience to Scioderm, in addition to his in depth transactional and public sector market experience. His personal drive, commitment and overall determination to find cures for rare diseases such as Pompe and Fabry...

2013-11-22 23:00:46

According to a review published on DailyGossip.org on November 21, Dr. Gary M. Levin has some amazing news for urticaria patients. Denver, Colorado (PRWEB) November 22, 2013 The Urticaria Cure by Dr. Gary Levin is the result of many years of experience, trial and experimentation. The review available on Daily Gossip Magazine indicates that this method was created by Dr. Gary with the main purpose to help patients cure chronic hives. To find out more about Dr. Gary M. Levin's latest...