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https://www.webmd.com/children/what-are-lysosomal-storage-disorders
Lysosomal storage disorders are a group of more than 50 rare diseases. They affect the lysosome -- a structure in your cells that breaks down substances such as proteins, carbohydrates, and old ...
http://www.authorstream.com/Presentation/phani.thanneru-1598552-drug-delivery-lysosomal-storage-diseases/
Drug Delivery to Lysosomal Storage Diseases - authorSTREAM Presentation. Slide 18: Enzyme Delivery across the Blood-Brain Barrier(BBB): 1.BBB is characterized by a layer endothelial cells, joined by tight junction 2. this restricts transport of bulk recombinant enzymes across brain Methods used to by pass the barrier are 1 .opening BBB by infusion of hypo osmotic solution (arabinose) into the ...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3969788/
Jun 01, 2012 · Strategies for Delivery of Therapeutics into the Central Nervous System for Treatment of Lysosomal Storage Disorders. ... The later two approaches are being pursued at the time by coupling therapeutic agents to affinity moieties and drug delivery systems capable of targeting these natural transport routes. This approach is particularly ...Author: Silvia Muro
http://vnips.in/Study%20Materials/Lysosomal%20Storage%20Diseases%20VNIPS.pdf
drug delivery to lysosomal storage diseases viswanadha institute of pharmaceutical sciences contents 1. introduction 2. lsd‟s 3. strategies for treatment or drug delivery of lsd‟s 4. carriers for treatment of lsd‟s 5. strategies for brain delivery 6. formulation aspects 7. current and …
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4881964/
With the aim to move into functional and efficacy studies, targeted to treatment of the brain disease in Lysosomal Storage Disorders, we here conducted, for the first time, several preliminary experiments in the murine models of MPS I and MPS II to explore the ability of g7-NPs to deliver across BBB a model drug (FITC-albumin), with a high ...Author: Marika Salvalaio, Laura Rigon, Daniela Belletti, Francesca D’Avanzo, Francesca Pederzoli, Barbara Ru...
https://www.nature.com/articles/s41598-019-55136-4
Dec 09, 2019 · Most lysosomal storage disorders affect the central nervous system. However, lysosomal enzymes do not cross the blood-brain barrier (BBB), and intravenous enzyme infusion is …Author: Ruben J. Boado, Jeff Zhiqiang Lu, Eric Ka-Wai Hui, Huilan Lin, William M. Pardridge
https://www.lysosomaltx.com/
Lysosomal Therapeutics Inc. (LTI) discovers and develops new therapies for patients with severe neurological diseases. Our strategy leverages the clinically-validated link between lysosome-based genetic disorders and neurodegenerative diseases to establish a unique and effective platform for …
https://www.sciencedirect.com/topics/medicine-and-dentistry/lysosomal-storage-disease
The lysosomal storage diseases are a clinically heterogenous group of inherited diseases, at least 50 in all, with a combined incidence of approximately one in 7000 births (Table 198-1).They present with protean manifestations, including significant involvement of the central and peripheral nervous systems. They are a challenge to the physician because signs and symptoms of these disorders may ...
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