Latest Rare diseases Stories
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
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,
ReportsnReports.com adds “OpportunityAnalyzer Amyotrophic Lateral Sclerosis (ALS) - Opportunity Analysis and Forecasts to 2018” to its store.
Talc Pleurodesis Offers Several Benefits Over Video-Assisted Method of Reducing Fluid Buildup in Lungs DALLAS (PRWEB) July 10, 2014 Researchers in the
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
- 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
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.
Approval delivers first pharmaceutical therapy for children and adults with ultra-rare, life-limiting disorder TORONTO, July 7, 2014 /CNW/ - BioMarin Pharmaceutical Inc.
Phase 3 Study Meets Primary Endpoint and Six Secondary Endpoints Across Multiple Disease-Related Abnormalities LEXINGTON, Mass., June 30, 2014 /PRNewswire/ --
WASHINGTON, June 29, 2014 /PRNewswire-USNewswire/ -- In work supported by The ALS Association, researchers have shown that increasing the clearance of misfolded protein from neurons improves