Current treatment options and novel nanotechnology-driven enzyme replacement strategies for lysosomal storage disorders

A Del Grosso, G Parlanti, R Mezzena… - Advanced Drug Delivery …, 2022 - Elsevier
Lysosomal storage disorders (LSDs) are a vast group of more than 50 clinically identified
metabolic diseases. They are singly rare, but they affect collectively 1 on 5,000 live births …

Role of nanotechnology for enzyme replacement therapy in lysosomal diseases. A focus on Gaucher's disease

L Martín-Banderas, MA Holgado… - Current Medicinal …, 2016 - ingentaconnect.com
Lysosomal storage diseases (LSDs) comprise a group of rare inherited chronic syndromes
that cause deficiency of specific native enzymes within the lysosomes. The macromolecular …

Treatment of lysosomal storage diseases: recent patents and future strategies

S Ortolano, I Vieitez, C Navarro… - Recent patents on …, 2014 - ingentaconnect.com
Lysosomal storage diseases (LSDs) are a group of rare genetic multisystemic disorders,
resulting in deficient lysosomal activity. These pathologies are characterized by progressive …

Enzyme replacement therapy: a review and its role in treating lysosomal storage diseases

M Li - Pediatric annals, 2018 - journals.healio.com
Lysosomal storage diseases (LSDs) are a heterogeneous group of genetic disorders
caused by defects in lysosomal function that lead to multiorgan system damage. Due to wide …

Recent developments in therapeutic approaches for lysosomal storage diseases

L Urbanelli, A Magini, A Polchi… - Recent Patents on …, 2011 - ingentaconnect.com
Genetic mutations that cause specific lysosomal protein deficiencies account for more than
45 Lysosomal Storage Diseases (LSDs), mostly pre-adult disorders which are associated …

Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives

M Solomon, S Muro - Advanced drug delivery reviews, 2017 - Elsevier
Lysosomes and lysosomal enzymes play a central role in numerous cellular processes,
including cellular nutrition, recycling, signaling, defense, and cell death. Genetic deficiencies …

Emerging therapies for neuropathic lysosomal storage disorders

JM Kelly, A Bradbury, DR Martin, ME Byrne - Progress in Neurobiology, 2017 - Elsevier
Abstract Approximately 1 in 5000–8000 children are born annually with a lysosomal storage
disease (LSD), which affects their cells' ability to break down naturally occurring substrates …

[HTML][HTML] Enzyme replacement therapies: what is the best option?

A Safary, MA Khiavi, R Mousavi, J Barar, MA Rafi - BioImpacts: BI, 2018 - ncbi.nlm.nih.gov
Despite many beneficial outcomes of the conventional enzyme replacement therapy (ERT),
several limitations such as the high-cost of the treatment and various inadvertent side effects …

Treatment perspectives for the lysosomal storage diseases

GA Grabowski - Expert Opinion on Emerging Drugs, 2008 - Taylor & Francis
Background: The therapy of the lysosomal storage diseases (LSDs) was developed by
supplying adequate amounts of the needed enzyme to affected individuals. This approach in …

Treatment options for lysosomal storage disorders: developing insights

CM van Gelder, AAM Vollebregt, I Plug… - Expert opinion on …, 2012 - Taylor & Francis
Introduction: Lysosomal storage disorders (LSDs) are clinically heterogeneous disorders
that result primarily from lysosomal accumulation of macromolecules in various tissues …