MPS disorders are associated with a wide spectrum of neurocognitive effects, from mild problems with attention and executive functions to progressive and degenerative …
M Tardieu, M Zérah, B Husson… - Human gene …, 2014 - liebertpub.com
Mucopolysaccharidosis type IIIA is a severe degenerative disease caused by an autosomal recessive defect of a gene encoding a lysosomal heparan-N-sulfamidase, the N …
M Beck - Developmental Medicine & Child Neurology, 2018 - Wiley Online Library
Over the past several years the number of treatments available for patients with lysosomal storage disorders has rapidly increased. Haematopoietic stem cell transplantation, enzyme …
A balanced metabolic profile is essential for normal human physiological activities. Disproportions in nutrition give rise to imbalances in metabolism that are associated with …
R Li, M Robinson, X Ding, T Geetha… - Journal of Food …, 2022 - Wiley Online Library
Neurodegenerative diseases are caused by the progressive loss of function or structure of nerve cells in the central nervous system. The most common neurodegenerative diseases …
F Andrade, L Aldámiz‐Echevarría… - Pediatrics …, 2015 - Wiley Online Library
Abstract Mucopolysaccharidosis type III (MPS III, Sanfilippo syndrome) is a lysosomal storage disorder, caused by a deficiency in one of the four enzymes involved in the …
WM Pardridge - Advanced Drug Delivery Reviews, 2022 - Elsevier
The majority of lysosomal storage diseases affect the brain. Treatment of the brain with intravenous enzyme replacement therapy is not successful, because the recombinant …
X Wang, Y Ma, Q Xu, AN Shikov, ON Pozharitskaya… - Phytomedicine, 2023 - Elsevier
Background Flavonoids and saponins are important bioactive compounds that have attracted wide research interests. This review aims to summarise the state of the art of the …
During the past decades, several therapeutic approaches have been developed and made rapidly available for many patients afflicted with lysosomal storage disorders (LSDs), inborn …