Pediatrics
Newborn screening tests approved
(HealthDay)—A set of screening tests designed to detect four rare metabolic disorders in newborns has been approved by the U.S. Food and Drug Administration.
Feb 6, 2017
0
0
News tagged with lysosomal storage disorder
Medical research
Research reveals strategy to potentially treat juvenile Batten disease
Researchers at Baylor College of Medicine, Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital and King's College London have discovered a treatment that improves the neurological symptoms in a ...
Feb 6, 2017
0
56
Genetics
Repurposed drugs may offer improved treatments for fatal genetic disorders
University of Rochester Medical Center researchers believe they have identified a potential new means of treating some of the most severe genetic diseases of childhood, according to a study in PLOS Biology. The diseases, ...
Dec 15, 2016
0
0
Genetics
Conquering the lysosomal storage disorders with genetic engineering
(Medical Xpress)—The motivation by fear, anger, and love to preserve self, kin, and others perches near the pinnacle of our existence. Triumph in the face of a seemingly insurmountable medical adversity by the creation ...
Genetics
Batten disease may benefit from gene therapy
In a study of dogs, scientists showed that a new way to deliver replacement genes may be effective at slowing the development of childhood Batten disease, a rare and fatal neurological disorder. The key may be to inject viruses ...
Nov 11, 2015
0
427
Medical research
NIH teams with industry to develop treatments for Niemann-Pick disease type C
Researchers from the National Institutes of Health have entered into an agreement with biotechnology company Vtesse, Inc., of Gaithersburg, Maryland, to develop treatments for Niemann-Pick disease type C (NPC) and other lysosomal ...
Jan 7, 2015
0
6
Neuroscience
Very early treatment may be key to combatting inherited metabolic disorder
A European Journal of Neuroscience study suggests that it is critical to treat lysosomal storage disorders early, before symptoms arise. These genetic disorders, which are caused by the malfunction of enzymes that normally ...
Aug 4, 2014
0
0
Medical research
Surprising culprit found in cell recycling defect
(Medical Xpress)—To remain healthy, the body's cells must properly manage their waste recycling centers. Problems with these compartments, known as lysosomes, lead to a number of debilitating and sometimes lethal conditions.
Feb 20, 2014
0
0
Medical research
New technology for more efficient treatment of Pompe disease, other metabolic disorders
VIB researchers from UGent and Vrije Universiteit Brussel , together with a team of the firm Oxyrane have developed a new technology that can lead to a more efficient and possibly also cheaper therapy for diseases such as ...
Nov 19, 2012
0
0
