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Full Prescribing Information (PDF)

General Physical Appearance Auditory System
Nervous System Ocular System
Skeletal System Cardiovascular System
Respiratory System Gastrointestinal System
Nervous System
Developmental Delay and Severe Mental Retardation
Patients with MPS I manifest a wide range of intellectual involvement. Some MPS I patients may suffer progressive and profound mental retardation, while others may show little or no intellectual dysfunction.[1] By 18 months, developmental delay is usually apparent. From this point on, patients generally do not progress in development but plateau for a number of years followed by a slow decline in intellectual capabilities.

Structural CNS Manifestations
In addition to the direct CNS effects of MPS I, hydrocephalus is common in MPS I patients. An associated increase in intracranial pressure may lead to brain compression, and rapidly increasing pressure may be the cause of acute developmental decline in some patients. Symptoms may be difficult to assess and progression can be insidious and is often under-appreciated. Lumbar puncture with opening pressure is a preferred method for assessing the degree of pressure elevation. Shunting procedures may be beneficial.[2] Another recognized complication is spinal cord compression, which can result from thickening of the dura or subluxation of vertebrae.

Peripheral Nervous System
Some patients with MPS I may have poor hand function, in part as a result of carpal tunnel syndrome. This syndrome is due to pressure on the median nerve as a result of thickened ligaments within the wrist. It may cause pain and numbness in the fingertips, but most patients lack these typical symptoms. [3][4][5] Diagnosis of carpal tunnel syndrome is often missed because its onset is insidious and often presents with few or no symptoms except thenar atrophy. Patients should be evaluated for this, as they may benefit from carpal tunnel release.


References:
1. Clarke LA. Clinical diagnosis of lysosomal storage diseases. In: Organelle Diseases. Clinical Features, Diagnosis, Pathogenesis and Management. Applegarth DA, Dimmick JE, Hall JG, editors. London: Chapman and Hall Medical; 1997. p. 37.

2. Neufeld EF, Muenzer J. The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D, Childs B, Kinzler KW, et al, editors. The Metabolic and Molecular Bases of Inherited Disease. Vol. III. 8th ed. New York: McGraw-Hill; 2001. p. 3427-8.

3. Wraith JE, Alani SM Carpal tunnel syndrome in the mucopolysaccharidoses and related disorders. Arch Dis Child 1990;65:962.

4. Haddad FS, Jones DH, Vellodi A., Kane N., Pitt MC Carpal tunnel syndrome in the mucopolysaccharidoses and the mucolipidoses. J Bone Joint Surg Br 1997;79:576.

5. Van Heest AE, House J, Krivit W, Walker K. Surgical treatment of carpal tunnel syndrome and trigger digits in children with mucopolysaccharide storage disorders. J Hand Surg (Am) 1998;23:236.


Region/Country
This site is intended for use in the United States. Please visit the Genzyme site for your country or region.
Even though people with MPS I have the same enzyme deficiency, they may experience a wide range of symptoms.
Information on MPS I disease is limited because of its rarity. Learn more about MPS I Registry, a global resource dedicated to improving the understanding of MPS I disease.

ALDURAZYME® (laronidase) 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.

 

Important Safety Information

 

WARNING

Risk of anaphylaxis.

Life-threatening anaphylactic reactions have been observed in some patients during ALDURAZYME infusions.  Therefore, appropriate medical support should be readily available when ALDURAZYME is administered.  Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions, and require additional monitoring.

 

Life-threatening anaphylactic reactions have been observed in some patients during or up to 3 hours after ALDURAZYME infusions. Reactions have included: respiratory failure, respiratory distress, stridor, tachypnea, bronchospasm, airway obstruction, hypoxia, hypotension, bradycardia, and urticaria. Interventions have included: resuscitation, mechanical ventilatory support, emergency tracheotomy, hospitalization, and treatment with inhaled beta-adrenergic agonists, epinephrine, and intravenous corticosteroids.

 

In clinical trials and postmarketing safety experience with ALDURAZYME, approximately 1% of patients experienced severe or serious allergic reactions. In patients with MPS I, pre-existing upper airway obstruction may have contributed to the severity of some reactions. Due to the potential for severe allergic reactions, appropriate medical support should be readily available when ALDURAZYME is administered. Because of the potential for recurrent reactions, some patients who experience initial severe reactions may require prolonged observation. The risks and benefits of re-administering ALDURAZYME following an anaphylactic or severe allergic reaction should be considered.

 

Patients with an acute illness at the time of ALDURAZYME infusion may be at greater risk for infusion-related reactions. Careful consideration should be given to the patient’s clinical status prior to administration of ALDURAZYME.

 

Patients should receive antipyretics and/or antihistamines prior to infusion. If an infusion reaction occurs, regardless of pretreatment, decreasing the infusion rate, temporarily stopping the infusion, and/or administration of additional antipyretics and/or antihistamines may ameliorate the symptoms.

 

The most common adverse reactions associated with ALDURAZYME treatment in the clinical studies were upper respiratory tract infection, rash, and injection site reaction  The most common adverse reactions requiring intervention were infusion-related reactions involving flushing, fever, headache, and rash. 

 

In postmarketing experience with ALDURAZYME, severe and serious infusion-related reactions have been reported, some of which were life-threatening.  The most frequently reported adverse reactions included: chills, vomiting, nausea, arthralgia, diarrhea, tachycardia, abdominal pain, blood pressure increased, and oxygen saturation decreased. 

 

Approximately 91% of patients treated with ALDURAZYME in clinical studies were positive for antibodies to laronidase. The clinical significance of antibodies to ALDURAZYME is not known, including the potential for product neutralization. Adverse events should be reported promptly to Genzyme Medical Information at 800-745-4447, option 2. ALDURAZYME is available by prescription only. To learn more, please see the full prescribing information (PDF) including boxed warning, visit www.ALDURAZYME.com or contact Genzyme at 1-800-745-4447.
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