ALDURAZYME® (laronidase) is a polymorphic variant of the human enzyme, (alpha)-L-iduronidase that is produced by recombinant DNA technology in a Chinese hamster ovary cell line. (alpha)-L-iduronidase (glycosaminoglycan (alpha)-L-iduronohydrolase, EC 126.96.36.199) is a lysosomal hydrolase that catalyses the hydrolysis of terminal (alpha)-L-iduronic acid residues of dermatan sulfate and heparan sulfate.
ALDURAZYME is indicated for patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms. The risks and benefits of treating mildly affected patients with the Scheie form have not been established.
ALDURAZYME has been shown to improve pulmonary function and walking capacity. ALDURAZYME has not been evaluated for effects on the central nervous system manifestations of the disorder.
Media Articles Related to Aldurazyme (Laronidase)
Researchers discover potential drug target for rare genetic disease Mucopolysaccharidosis I (MPS I)
Source: Bones / Orthopedics News From Medical News Today [2013.11.18]
Medical researchers at the University of Alberta have discovered the structure of a potential drug target for a rare genetic disease, paving the way for an alternative treatment for the condition.Faculty of Medicine & Dentistry researcher Michael James and his team recently published their findings about MPS I (Mucopolysaccharidosis I) in the peer-reviewed journal, Nature Chemical Biology.
Published Studies Related to Aldurazyme (Laronidase)
A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I. [2009.01]
Recombinant human alpha-l-iduronidase (Aldurazyme), laronidase) is approved as an enzyme replacement therapy to treat the lysosomal storage disorder, mucopolysaccharidosis type I (MPS I) at a dose of 0.58 mg/kg by once-weekly intravenous infusion. To assess whether alternate dosing regimens might provide a better reduction in lysosomal storage, a 26-week, randomized, open-label, multinational dose-optimization trial was conducted...
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. [2009.01]
OBJECTIVE: Our goal was to evaluate the long-term safety and efficacy of recombinant human alpha-l-iduronidase (laronidase) in patients with mucopolysaccharidosis I... CONCLUSIONS: This trial demonstrates the long-term clinical benefit and safety of laronidase in attenuated patients with mucopolysaccharidosis I and highlights the magnitude and chronology of treatment effects. Prompt diagnosis and early treatment will maximize treatment outcomes.
Laronidase for treating mucopolysaccharidosis type I. [2007.09.30]
Mucopolysaccharidoses are a group of inherited metabolic diseases caused by the absence or deficiency of the lysosomal enzymes that are needed for breaking down glycosaminoglycans (GAGs).Laronidase seems to be a promising agent for treating mucopolysaccharidosis type I, as shown by the reduction in the urinary excretion of GAGs and the associated improvements in vital capacity and in the performance of defined physical tasks.
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). [2004.05]
OBJECTIVE: To confirm the efficacy and safety of recombinant human alpha-L-iduronidase (laronidase) in patients with mucopolysaccharidosis I (MPS I)... CONCLUSIONS: In patients with MPS I, laronidase significantly improves respiratory function and physical capacity, reduces glycosaminoglycan storage, and has a favorable safety profile.
Laronidase replacement therapy improves myocardial function in mucopolysaccharidosis I. [2011.07]
We assessed whether laronidase (recombinant human alpha-L-iduronidase) replacement therapy could improve left ventricular (LV) myocardial function in a 49-year-old woman with mucopolysaccharidosis I (MPS I) and valvular heart disease...
Clinical Trials Related to Aldurazyme (Laronidase)
Immune Tolerance Study With Aldurazyme« (Laronidase) [Recruiting]
The purpose of this study is to see if treatment with an antigen-specific immunosuppressive
can decrease or stop an antibody response to laronidase during enzyme replacement therapy
with Aldurazyme in severe MPS I (Mucopolysaccharidosis I) patients.
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme´┐Ż Treated Patients [Recruiting]
The purpose of this study is to determine whether the development of antibodies to
Aldurazyme« (laronidase) in patients with MPS I receiving Aldurazyme« (laronidase) impairs
the clearance of GAG substrate.
A Study of the Effect of Aldurazyme´┐Ż (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants [Recruiting]
The purpose of this study is to determine if laronidase is present in the breast milk of
post-partum women receiving Aldurazyme« (laronidase) and the effects of Aldurazyme
(laronidase) on the growth, development, and immunologic response of their breastfed
A Dose-Optimization Study of Aldurazyme« (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease [Completed]
The main purpose of this study is to evaluate differences in the pharmacodynamic response of
4 Aldurazyme« (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I)
disease through measuring urinary GAG levels, liver volume, and functional tests (6-minute
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme« (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients [Completed]
This study is being conducted to collect additional long-term efficacy and safety data of
Aldurazyme« (Laronidase) patients with MPS I disease. Patients who were previously enrolled
in the phase 3 double-blind study will be enrolled in this study.
Reports of Suspected Aldurazyme (Laronidase) Side Effects
Respiratory Disorder (8),
Spinal Cord Compression (7),
Infusion Related Reaction (7),
Device Related Infection (5),
Pallor (5), more >>