Idursulfase for the treatment of mucopolysaccharidosis II

Expert Opinion on Pharmacotherapy
Lorne A Clarke

Abstract

Human recombinant proteins are being used to treat an increasing number of disorders. Advances in the large scale production of recombinant proteins and the understanding of glycosylation and its importance for protein targeting and function have led to the development of recombinant enzyme-replacement regimens for a number of human lysosomal storage diseases. The latest addition to this group is mucopolysaccharidosis II or Hunter syndrome. Purified human recombinant idursulfase has been shown to alter disease manifestations in individuals with Hunter syndrome. The recent approval in the US, Europe, Canada and Japan of idursulfase for the treatment of Hunter syndrome introduces the first pharmacologic agent and indeed the first specific treatment directed towards this devastating genetic disease.

References

Jan 1, 1992·Advances in Experimental Medicine and Biology·C Freeman, J Hopwood
Nov 1, 1990·Proceedings of the National Academy of Sciences of the United States of America·P J WilsonJ J Hopwood
Jan 1, 1986·The Journal of Clinical Investigation·S Kornfeld
Sep 1, 1993·Genomics·P J WilsonC P Morris
Jan 1, 1993·Journal of Inherited Metabolic Disease·S U Walkley, P A March
Jul 1, 1996·The Journal of Pediatrics·E J McKinnisC R Scott
Jan 26, 1999·JAMA : the Journal of the American Medical Association·P J MeikleW F Carey
Jul 10, 1999·Journal of Inherited Metabolic Disease·A VellodiJ E Wraith
Feb 15, 2001·The New England Journal of Medicine·E D KakkisE F Neufeld
Nov 11, 2003·American Journal of Medical Genetics. Part a·John NelsonLawrence Greed
Jun 23, 2004·Seminars in Cell & Developmental Biology·Steven U Walkley
Oct 27, 2004·The Biochemical Journal·Emma Parkinson-LawrenceDoug Brooks
Aug 17, 2006·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Joseph MuenzerAlan Kimura
Dec 23, 2006·Molecular Genetics and Metabolism·Joseph MuenzerAlan Kimura
Apr 27, 2007·Molecular Genetics and Metabolism·Antony R GarciaJustin C Lamsa
Jun 15, 2007·Cellular and Molecular Life Sciences : CMLS·D Ghosh

❮ Previous
Next ❯

Citations

Sep 6, 2012·Archivum Immunologiae Et Therapiae Experimentalis·Zyta Banecka-MajkutewiczGrzegorz Węgrzyn
Jun 4, 2011·Genetics and Molecular Biology·Roberto GiuglianiAna Maria Martins
Feb 11, 2014·Cardiovascular Pathology : the Official Journal of the Society for Cardiovascular Pathology·Elizabeth BraunlinJ Carlos Manivel
Jul 30, 2014·Genetics and Molecular Biology·Roberto GiuglianiHernán Amartino
May 1, 2020·Frontiers in Molecular Biosciences·Jacob M FavretDaesung Shin
Mar 11, 2009·British Journal of Nursing : BJN·Catherine LittleChris Hendriksz

❮ Previous
Next ❯

Related Concepts

Trending Feeds

COVID-19

Coronaviruses encompass a large family of viruses that cause the common cold as well as more serious diseases, such as the ongoing outbreak of coronavirus disease 2019 (COVID-19; formally known as 2019-nCoV). Coronaviruses can spread from animals to humans; symptoms include fever, cough, shortness of breath, and breathing difficulties; in more severe cases, infection can lead to death. This feed covers recent research on COVID-19.

Blastomycosis

Blastomycosis fungal infections spread through inhaling Blastomyces dermatitidis spores. Discover the latest research on blastomycosis fungal infections here.

Nuclear Pore Complex in ALS/FTD

Alterations in nucleocytoplasmic transport, controlled by the nuclear pore complex, may be involved in the pathomechanism underlying multiple neurodegenerative diseases including Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Here is the latest research on the nuclear pore complex in ALS and FTD.

Applications of Molecular Barcoding

The concept of molecular barcoding is that each original DNA or RNA molecule is attached to a unique sequence barcode. Sequence reads having different barcodes represent different original molecules, while sequence reads having the same barcode are results of PCR duplication from one original molecule. Discover the latest research on molecular barcoding here.

Chronic Fatigue Syndrome

Chronic fatigue syndrome is a disease characterized by unexplained disabling fatigue; the pathology of which is incompletely understood. Discover the latest research on chronic fatigue syndrome here.

Evolution of Pluripotency

Pluripotency refers to the ability of a cell to develop into three primary germ cell layers of the embryo. This feed focuses on the mechanisms that underlie the evolution of pluripotency. Here is the latest research.

Position Effect Variegation

Position Effect Variagation occurs when a gene is inactivated due to its positioning near heterochromatic regions within a chromosome. Discover the latest research on Position Effect Variagation here.

STING Receptor Agonists

Stimulator of IFN genes (STING) are a group of transmembrane proteins that are involved in the induction of type I interferon that is important in the innate immune response. The stimulation of STING has been an active area of research in the treatment of cancer and infectious diseases. Here is the latest research on STING receptor agonists.

Microbicide

Microbicides are products that can be applied to vaginal or rectal mucosal surfaces with the goal of preventing, or at least significantly reducing, the transmission of sexually transmitted infections. Here is the latest research on microbicides.

© 2021 Meta ULC. All rights reserved