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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.
http://www.authorstream.com/Presentation/phani.thanneru-1598552-drug-delivery-lysosomal-storage-diseases/
Storage Drug Diseases Delivery Lysosomal *You can't enter more than 5 tags. Enter one or more tags separated by comma or enter.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3969788/
Jun 01, 2012 · 1.2. General Strategies for Treatment of Lysosomal Storage Disorders. The development of effective therapies for LSDs has been historically hampered due to the low incidence of these illnesses, which ranges between 1:25,000 to 1:250,000 for each individual disease, with 1:5,000 to 1:7,000 affected newborns as a group . This fact made it specially difficult to attract significant support …Author: Silvia Muro
https://www.goodrx.com/lysosomal-storage-disease/drugs
SEBELIPASE ALFA is a replacement enzyme used to treat patients with lysosomal acid lipase (LAL) deficiency (also known as Wolman disease). It is not a cure. GALSULFASE is an enzyme replacement. It is used to treat the symptoms of mucopolysaccharidosis VI (also known as MPS VI or Maroteaux-Lamy syndrome).
https://www.scilit.net/article/b9712929cd18ea07ea54c5465e720748
Between 1983 and 2019, 124 orphan drug designations were granted by the FDA for compounds intended to treat 28 lysosomal storage diseases. Orphan drug designations focused on Gaucher disease (N = 16), Pompe disease (N = 16), Fabry disease (N = 10), MPS II (N = 10), MPS I (N = 9), and MPS IIIA (N = 9), and included enzyme replacement therapies ...
https://www.ncbi.nlm.nih.gov/pubmed/30315863
Nanotechnology may be a promising way to circumvent the drawbacks of the current therapies for lysosomal diseases. The blood circulation time and bioavailability of the enzymes or drugs could be improved by inserting them in nanocarriers, which could decrease and/or avoid the need of frequent intravenous infusions along with the minimization or elimination of associated immunogenic responses.Author: Bruna Donida, Bárbara Tauffner, Marco Raabe, Maira F. Immich, Marcelo A. de Farias, Diego de Sá Cout...
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