Sentences with phrase «for lysosomal storage»
Penati R *, Fumagalli F *, Calbi V, Bernardo ME, Aiuti A. Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I. J Inherit Metab Dis.
http://research.hsr.it/ Not exact matches
The drug candidate is intended for the treatment of subjects with neuronal ceroid lipofuscinosis, aka Batten Disease, a fatal lysosomal storage disease that primarily affects the nervous system in children.
Genzyme has long been known for our expertise in the class of rare genetic diseases known as lysosomal storage disorders (LSDs).
Enhanced delivery of α - glucosidase for Pompe disease by ICAM -1-targeted nanocarriers: comparative performance of a strategy for three distinct lysosomal storage disorders.
The research establishes a new model for studying lysosomal storage disorders and suggests that GM2 accumulation not only causes neurodegeneration but also may disrupt neurodevelopment prior to birth.
Dr. Jim ’83 and Anne ’80 Gardner, who own Gardner Animal Hospital in Eufaula, learned through social media about the lifesaving research being conducted at the college's Scott - Ritchey Research Center for GM1 gandliosidosis, a lysosomal storage disease that attacks the brain and spinal cord, and is a progressive and degenerative condition that is always fatal in children.