Mucopolysaccharidosis Type 1 (MPS I, Hurler Syndrome) Treatments
Find Mucopolysaccharidosis Type 1 (MPS I, Hurler Syndrome) Treatments
Brand Name
Aldurazyme
Generic Name
Laronidase
View Brand Information FDA approval date: April 30, 2003
Classification: Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme
Form: Injection
What is Aldurazyme (Laronidase)?
ALDURAZYME ® is indicated for the treatment of: adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I and patients with the Scheie form of MPS I who have moderate to severe symptoms. ALDURAZYME is a hydrolytic lysosomal glycosaminoglycan -specific enzyme indicated for the treatment of adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I and for the treatment of patients with the Scheie form of MPS I who have moderate to severe symptoms. Limitations of Use: The risks and benefits of treating mildly affected patients with the Scheie form have not been established. ALDURAZYME has not been evaluated for effects on the central nervous system manifestations of the disorder. Limitations of Use The safety and effectiveness of treating mildly affected patients with the Scheie form have not been established. The effect of ALDURAZYME on central nervous system manifestations of the disorder has not been determined.
Approved To Treat
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Related Clinical Trials
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Summary: For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.