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Latest Rare diseases Stories

2014-01-30 12:28:16

TUCSON, Ariz. and CAMBRIDGE, Mass., Jan. 30, 2014 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association (MDA) and Augie Nieto, co-chairman of MDA's ALS division, together with his wife, Lynne, today announced they are transitioning management of the successful "Augie's Quest" campaign from MDA to the ALS Therapy Development Institute (ALS TDI). The transition will be occurring in the first quarter of 2014 as the two organizations continue to support Augie's Quest initiatives....

2014-01-28 09:12:25

Kennedy Krieger researchers develop valid and accurate model for FSHD Researchers at the Kennedy Krieger Institute recently announced study findings showing the successful development of a humanized preclinical model for facioscapulohumeral muscular dystrophy (FSHD), providing scientists with a much needed tool to accelerate novel therapeutic research and development. Published in Human Molecular Genetics, the study outlines the validity of a unique model that, for the first time,...

2014-01-24 23:02:29

Recent scientific development represents progress in FSHD research. Lexington, Mass. (PRWEB) January 24, 2014 As published online today in Human Molecular Genetics, support from the FSH Society, a patient-driven nonprofit, has enabled people with facioscapulohumeral muscular dystrophy (FSHD) to donate muscle tissue, which scientists have succeeded in grafting into mice, providing a new tool for conquering this devastating muscle-wasting disease. Among the most common forms of muscular...

2014-01-20 10:36:30

What causes brain damage and inflammation in severe cases of Gaucher disease? Little is known about the events that lead to brain pathology in some forms of the disease, and there is currently no treatment available – a bleak outlook for sufferers and their families. Now, scientists at the Weizmann Institute of Science have discovered a new cellular pathway implicated in Gaucher disease. Their findings, published today in Nature Medicine, may offer a new therapeutic target for the...

2014-01-16 23:31:02

Cellular Life Research Center has awarded it's Advanced Research Recognition award to PIMTforLife. In clinical trials, patients taking PIMTforLife, an all-natural anti-aging tablet, have shown a dramatic reverse in signs of aging in as little time as three weeks. St. Petersburg, FL (PRWEB) January 16, 2014 PIMTforLife is the number one anti-aging tablet recognized by the Cellular Life Research Center for its unique and highly effective utilization of the PIMT enzyme and is also now...

2014-01-16 16:24:09

AUSTIN, Texas, Jan. 16, 2014 /PRNewswire/ -- XBiotech announced today that it has launched a Phase II study to treat a rare but debilitating disorder, Pyoderma Gangrenosum (PG). This study is based on efficacy seen with anti-IL-1 therapeutic antibody in previous dermatological clinical studies such as psoriasis and acne. PG is expected to be considered an "orphan indication" by the FDA, facilitating expedited clinical development to market. Dr. Armand Cognetta, lead clinical...

2014-01-16 12:31:54

New Center Expected to Attract Patients around the Nation PHOENIX, Jan. 16, 2014 /PRNewswire/ -- Barrow Neurological Institute at St. Joseph's Hospital and Medical Center in Phoenix have announced the opening of a new groundbreaking center expected to bring Arizona to the forefront of research and medical treatment for one of the world's most devastating and debilitating diseases - ALS, commonly known as Lou Gehrig's disease. The Gregory W. Fulton ALS and Neuromuscular Disorders...

2014-01-14 12:24:58

WASHINGTON, Jan. 14, 2014 /PRNewswire-USNewswire/ -- Men's Senior Baseball League/Men's Adult Baseball League (MSBL/MABL), the country's premier amateur baseball league for adults 18 years of age and older, has partnered with The ALS Association, in the fight against amyotrophic lateral sclerosis (ALS). (Logo: http://photos.prnewswire.com/prnh/20131211/MM32178LOGO) ALS, commonly known as Lou Gehrig's Disease, is a progressive neurodegenerative disease that affects nerve cells in...

Gene Variation In Mild Cognitive Impairment Associated With Brain Atrophy
2014-01-14 10:09:03

Radiological Society of North America The presence of a gene variant in people with mild cognitive impairment (MCI) is associated with accelerated rates of brain atrophy, according to a new study published online in the journal Radiology. The study focused on the gene apolipoprotein E (APOE), the most important genetic factor known in non-familial Alzheimer's disease (AD). APOE has different alleles, or gene variations, said the study's senior author, Jeffrey R. Petrella, M.D.,...

2014-01-03 12:23:32

TOWSON, Md., Jan. 3, 2014 /PRNewswire/ -- The Immune Deficiency Foundation (IDF), the national patient organization for persons with primary immunodeficiency diseases, commends the state of Washington for their decision to add Severe Combined Immune Deficiency (SCID) to the list of conditions that all newborns in the state are screened for at birth effective January 1, 2014. SCID is commonly known as "bubble boy disease" after David Vetter, a boy who lived in a germ-free bubble for...


Word of the Day
bellycheer
  • Good cheer; viands.
  • To revel; to feast.
The word 'bellycheer' may come from 'belle cheer', "good cheer".
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