Latest Glycogen storage disease type II Stories
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.
Three neuromuscular diseases are being considered for inclusion in nationwide screening. Tucson, AZ (PRWEB) September 27, 2013 Pediatrician and medical
Kids with Pompe disease fail because of a missing enzyme, GAA, that leads to dangerous sugar build-up, which affects muscles and movement.
Researchers set their eyes on a new treatment! Patients that have been affected by melanoma of the eye, ocular or uveal melanoma, die within an average of two to four months once the disease has spread to the liver due to ineffective treatment.
NOVATO, Calif., July 28, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced an update on the GALNS Phase 1/2 extension study (MOR-100) in which patients have continued treatment on an ongoing basis.
CRANBURY, N.J., May 4, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD), a biopharmaceutical company at the forefront of developing therapies for rare diseases, today announced financial results for the first quarter ended March 31, 2011.
- To swell, as grain or wood with water.