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Latest Rare diseases Stories

2014-07-22 08:32:03

- Neurotrope enters into world-wide, exclusive license agreement with Mount Sinai; Files related provisional patent for Bryostatin-1 - PLANTATION, Fla., July 22, 2014 /PRNewswire/ -- Neurotrope Bioscience, Inc., a subsidiary of Neurotrope, Inc. (OTCQB: NTRP) has signed a world-wide, exclusive license agreement with the Icahn School of Medicine at Mount Sinai (Mount Sinai) to utilize its proprietary information and data package for the use of Bryostatin-1 in the treatment of...

2014-07-18 08:24:03

DUBLIN, July 18, 2014 /PRNewswire/ -- NOT FOR RELEASE, PUBLICATION OR DISTRIBUTION (IN WHOLE OR IN PART) IN, INTO OR FROM ANY JURISDICTION WHERE TO DO SO WOULD CONSTITUTE A VIOLATION OF THE RELEVANT LAWS OF SUCH JURISDICTION. Increases Non GAAP diluted earnings per ADS guidance to low-to-mid thirty percent growth in 2014. Shire (LSE: SHP, NASDAQ: SHPG) announces unaudited results for the three months to June 30, 2014....

2014-07-17 16:25:53

Publication in Science Unveils Novel, Diverse and Separate Biological Functions That Can Be Harnessed Therapeutically to Mediate a Wide Range of Human Disease SAN DIEGO and HONG KONG, July 17, 2014 /PRNewswire/ -- aTyr Pharma, an innovative rare disease therapeutics enterprise, and its subsidiary in Hong Kong, Pangu Biopharma (aTyr), announced the discovery of a new class of human proteins with therapeutic relevance for a wide range of diseases. The findings unveiled today in a...

2014-07-16 23:03:05

ReportsnReports.com adds “OpportunityAnalyzer Amyotrophic Lateral Sclerosis (ALS) - Opportunity Analysis and Forecasts to 2018” to its store. Patent Expiration of Rilutek to Cause Negative Growth in Amyotrophic Lateral Sclerosis Treatment Market by 2018. Dallas, TX (PRWEB) July 16, 2014 The amyotrophic lateral sclerosis (ALS) treatment market value will decline from $64 million in 2013 to $38 million by 2018, at a negative Compound Annual Growth Rate (CAGR) of 10.05%, according to...

2014-07-10 23:09:39

Talc Pleurodesis Offers Several Benefits Over Video-Assisted Method of Reducing Fluid Buildup in Lungs DALLAS (PRWEB) July 10, 2014 Researchers in the United Kingdom have found that a procedure that uses talc to help seal the lining of the lungs in patients suffering from pleural mesothelioma is superior to a video-assisted surgical procedure in reducing hospital stays and the occurrence of complications, the mesothelioma law firm of Baron and Budd reports. The results of the research...

2014-07-08 16:25:25

TOWSON, Md., July 8, 2014 /PRNewswire/ -- With the recent addition of newborn screening for Severe Combined Immune Deficiency (SCID) in New Jersey and Illinois, approximately two-thirds of all babies born in the U.S. will be screened for this deadly disease. SCID is a primary immunodeficiency disease where affected infants lack the T-cells and antibody immunity that help fight infections from a wide array of viruses, bacteria and fungi, leaving these infants susceptible to serious,...

2014-07-07 16:24:20

- MPS IV A patients and families urge swift, publicly funded access to life-altering drug VIMIZIM(TM) - VANCOUVER, July 7, 2014 /CNW/ - The Canadian Society for Mucopolysaccharide & Related Diseases Inc. (Canadian MPS Society) is thrilled by Health Canada's recent approval of VIMIZIM(TM) (elosulfase alfa), the first and only pharmaceutical treatment option available for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Mucopolysaccharidosis IVA (MPS...

2014-07-07 16:24:11

NEW YORK, July 7, 2014 /PRNewswire/ -- Lysosomal storage disorders are a group of approximately 50 rare inherited metabolic disorders resulting from defects in lysosomal function. These severe, chronic genetic diseases have unmet medical needs, and include Fabry disease, Gaucher disease, Parkinson's disease, Pompe disease, and Tay-Sachs disease. The market for current drug therapies to address these disorders is estimated to exceed $3 billion. Amicus Therapeutics, Inc. (NASDAQ:...

2014-07-07 08:27:54

Approval delivers first pharmaceutical therapy for children and adults with ultra-rare, life-limiting disorder TORONTO, July 7, 2014 /CNW/ - BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that Health Canada has approved VIMIZIM(TM)(elosulfase alfa) for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis IVA (MPS IVA), also known as Morquio A syndrome. The approval makes VIMIZIM the first and only pharmaceutical...

2014-06-30 16:26:45

Phase 3 Study Meets Primary Endpoint and Six Secondary Endpoints Across Multiple Disease-Related Abnormalities LEXINGTON, Mass., June 30, 2014 /PRNewswire/ -- Synageva BioPharma Corp. (Synageva) (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, announced today that the global, randomized, double-blind, placebo-controlled Phase 3 ARISE trial of sebelipase alfa in 66 children and adults with lysosomal acid lipase deficiency (LAL...


Word of the Day
penuche
  • A fudgelike confection of brown sugar, cream or milk, and chopped nuts.
'Penuche' is a variant of 'panocha,' a coarse grade of sugar made in Mexico. 'Panocha' probably comes from the Spanish 'panoja, panocha,' ear of grain.
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