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Naglazyme (Galsulfase) - Summary

 
 



NAGLAZYME SUMMARY

NAGLAZYME (galsulfase) is a normal variant form of the polymorphic human enzyme, N-acetylgalactosamine 4-sulfatase that is produced by recombinant DNA technology in a Chinese hamster ovary cell line. N-acetylgalactosamine 4-sulfatase (glycosaminoglycan N-acetylgalactosamine 4-sulfatase, EC 3.1.6.12) is a lysosomal hydrolase that catalyzes the cleavage of the sulfate ester from terminal N-acetylgalactosamine 4-sulfate residues of glycosaminoglycans (GAG) chondroitin 4-sulfate and dermatan sulfate.

NAGLAZYME is indicated for patients with Mucopolysaccharidosis VI (MPS VI). NAGLAZYME has been shown to improve walking and stair-climbing capacity.


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NEWS HIGHLIGHTS

Published Studies Related to Naglazyme (Galsulfase)

Thrombocytopenia associated with galsulfase treatment. [2011.07]
Mucopolysaccharidosis type VI (MPS VI), or Maroteaux-Lamy syndrome, is a lysosomal storage disorder that results from a deficiency of the enzyme N-acetylgalactosamine-4-sulfatase or arylsulfatase B (ASB). It is a relatively rare disorder, with an estimated incidence of 1 in 248,000 to 1 in 300,000.Additionally, with this report, we want to share our approach for this case.

Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI): a single dose of galsulfase further reduces urine glycosaminoglycans after hematopoietic stem cell transplantation. [2010.12]
BACKGROUND: Maroteaux-Lamy syndrome, or mucopolysaccharidosis (MPS) type VI, is the autosomal recessive lysosomal disorder resulting from deficient N-acetylgalactosamine 4-sulfatase (ARSB) and the consequent accumulation of glycosaminoglycan (GAG). Patients fully engrafted after hematopoietic stem cell transplantation (HSCT) demonstrate several indicators of metabolic correction such as reduction in liver size, absence of hepatic ultrastructural pathology, and patients do not develop cervical cord compression. Engrafted patients demonstrate reduction in urine GAG achieving near-normal levels. HYPOTHESIS: We presumed that HSCT engraftment from a normal individual would provide sufficient systemic enzyme to accomplish maximal metabolic correction, and that no additional benefit would accrue from additional therapy such as with intravenous recombinant human ARSB protein, galsulfase... CONCLUSIONS: Somewhat surprisingly, the biomarker urine GAG was significantly reduced after a single treatment of intravenous galsulfase thus suggesting that supplemental enzyme replacement therapy might improve the clinical outcome for donor-engrafted patients with MPS VI. Copyright (c) 2010. Published by Elsevier Inc.

Enzyme replacement therapy with galsulfase for mucopolysaccharidosis VI: clinical facts and figures. [2010.09]
Mucopolysaccharidosis VI (MPS VI) is an inheritable, clinically heterogeneous lysosomal storage disorder that develops due to a deficiency in the arylsulfatase B (ASB) enzyme. This deficiency impairs the stepwise degradation of glycosaminoglycans (GAGs) resulting in the accumulation of partially degraded GAGs in tissues and organs throughout the body...

Reversed papilledema in an MPS VI patient with galsulfase (Naglazyme) therapy. [2009.08]
MPS VI (mucopolysaccharidosis VI, known as Maroteaux-Lamy syndrome) is a multi-systemic inherited disease, resulting from a deficiency of N-acetylgalactosamine-4-sulfatase, causing accumulation of the glycosaminoglycan (GAG) dermatan sulfate in all tissues.

A systematic review of new advances in the management of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): focus on galsulfase. [2009]
INTRODUCTION: Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) is an autosomal recessive lysosomal storage disorder, characterized primarily by skeletal dysplasia and joint contracture. It is caused by a deficiency of N-acetylgalactosamine-4-sulfatase (arylsulfatase B), for which a recombinant formulation (galsulfase) is available as replacement therapy. OBJECTIVE: To evaluate the effectiveness and safety of galsulfase compared to placebo or no interventions, for treating MPS VI. We also considered studies evaluating different doses of galsulfase... CONCLUSIONS: There is some evidence to support the use of galsulfase in the treatment of MPS VI; however due to the very low quantity of included studies we could not analyze it in an appropriate way. This review highlights the need for continued research into the use of enzyme replacement therapy for MPS VI.

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Clinical Trials Related to Naglazyme (Galsulfase)

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome [Not yet recruiting]
This is a single center study in which NaglazymeŽ will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic transplant.

A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI [Completed]
The purpose of the study is to evaluate the safety and efficacy of two dose levels of Naglazyme in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) [Recruiting]

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 [Completed]
There is limited information on the long-term effects of treating patients with MPS VI with NaglazymeŽ and limited data on the natural history of treated and untreated MPS VI patients. The Re-survey Study ASB-00-03 will assist in understanding the effects of long-term Naglazyme treatment and the natural history of the MPS VI patient population.

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Reports of Suspected Naglazyme (Galsulfase) Side Effects

Pneumonia (29)Spinal Cord Compression (28)Convulsion (17)Death (7)Bronchospasm (7)Hydrocephalus (6)Infusion Related Reaction (6)Cervical Cord Compression (6)Sleep Apnoea Syndrome (6)Product Quality Issue (5)more >>


Page last updated: 2011-12-09

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