The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities. The objectives of the Registry are: * To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase) * To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I * To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care
The MPS I Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact: * In Asia-Pacific - Vivian Liu, +65-6431-2548, Vivian.liu@genzyme.com * In Europe - +31-35-699-1232, europe@mpsiregistry.com * In Latin America - +617-591-5500, help@mpsiregistry.com * In North America - +617-591-5500, help@mpsiregistry.com
Study Type
OBSERVATIONAL
Enrollment
1,500
Phoenix Children's Hospital- Site Number : 840003
Phoenix, Arizona, United States
RECRUITINGArkansas Childrens Hospital- Site Number : 840109
Little Rock, Arkansas, United States
RECRUITINGSouthern California Permanente Medical Group- Site Number : 840108
Los Angeles, California, United States
RECRUITINGUSC Health Sciences Center Dept of Genetics- Site Number : 840082
Los Angeles, California, United States
To evaluate the long-term effectiveness of Aldurazyme
Time frame: Approximately 17 Years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Children's Hospital Oakland- Site Number : 840029
Oakland, California, United States
COMPLETEDChildren's Hospital of Orange County- Site Number : 840074
Orange, California, United States
RECRUITINGStanford Unviersity MC Dept of Genetics- Site Number : 840022
Palo Alto, California, United States
RECRUITINGUC Davis MIND Institute- Site Number : 840010
Sacramento, California, United States
RECRUITINGLoma Linda University Children's Hospital- Site Number : 840117
San Bernardino, California, United States
RECRUITINGUniversity of California at San Diego- Site Number : 840007
San Diego, California, United States
COMPLETED...and 170 more locations