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Full Prescribing Information (PDF)

MPS I is a progressive, debilitating, and often life-threatening disease. Over time, the enzyme deficiency and resulting accumulation of GAG (dermatan sulfate and heparan sulfate) in tissues and cells has progressively debilitating and often fatal effects, usually due to obstructive airway disease, respiratory infection, or cardiac complications. In the most severe cases of MPS I, death usually occurs by age 10 although some patients may have a normal life span.[1]

Clinical symptoms are heterogeneous and are progressively limiting in nature. A few examples of disease progression include:

Physical appearance of some infants with MPS I may appear normal at birth, but as early as three months of age, parents may notice changes in the child’s facial features.[2] The characteristic facial features of MPS I include short noses, flat faces, prominent foreheads, and large heads, which tend to be longer than normal from front to back (scaphocephalism).[3]

Developmental delay is usually apparent in patients with the severe form of MPS I by 12 to 24 months of age, with a maximum functional age of two to four years. This is followed by slowed development and progressive regression in developmental skills until death. [4]

Skeletal manifestations occur early in life, by about six months of age, in patients at the severe end of the spectrum. It is common to observe mild bone abnormalities (detected by radiological methods), particularly with hip abnormalities, ovoid vertebrae, and widening of the ribs. Other skeletal abnormalities include a poorly formed pelvis, a gibbus deformity of the back, and shortened phalanges.[4] Eventually, progressive skeletal dysplasia involving all bones is seen in all types of MPS I.




References:
1. 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.

2. Cleary MA, Wraith JE. The presenting features of mucopolysaccharidosis type IH (Hurler syndrome). Acta Paediatr. 1995;84:337-339.

3. Scheie HG, Hambrick GW, Barness LA. A newly recognized forme fruste of Hurler’s disease (Gargoylism). American Journal of Ophthalmology 1962;53(5):755.

4. 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.
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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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