Latest Rare diseases Stories
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
LYNBROOK, N.Y., June 25, 2014 /PRNewswire/ -- BioSpecifics Technologies Corp.
Computer analysis of photographs could help doctors diagnose which condition a child with a rare genetic disorder has, say Oxford University researchers.
DUBLIN, June 23, 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