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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1294-8266 | Registry Identifier | ICTRP |
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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:
The MPS I Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:
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| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the long-term effectiveness of Aldurazyme | Approximately 17 Years |
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Inclusion Criteria:
Exclusion Criteria:
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All Patients with MPS I
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Genzyme, a Sanofi Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix Children's Hospital- Site Number : 840003 | Phoenix | Arizona | 85016 | United States | ||
| Arkansas Children's Hospital- Site Number : 840109 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28892147 | Background | Viskochil D, Muenzer J, Guffon N, Garin C, Munoz-Rojas MV, Moy KA, Hutchinson DT. Carpal tunnel syndrome in mucopolysaccharidosis I: a registry-based cohort study. Dev Med Child Neurol. 2017 Dec;59(12):1269-1275. doi: 10.1111/dmcn.13545. Epub 2017 Sep 11. | |
| 25214650 | Background | Arn P, Bruce IA, Wraith JE, Travers H, Fallet S. Airway-related symptoms and surgeries in patients with mucopolysaccharidosis I. Ann Otol Rhinol Laryngol. 2015 Mar;124(3):198-205. doi: 10.1177/0003489414550154. Epub 2014 Sep 11. |
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| Little Rock |
| Arkansas |
| 72202 |
| United States |
| Southern California Permanente Medical Group- Site Number : 840108 | Los Angeles | California | 90027 | United States |
| USC Health Sciences Center Dept of Genetics- Site Number : 840082 | Los Angeles | California | 90033 | United States |
| UCSF Benioff Children's Hospital - Oakland- Site Number : 840029 | Oakland | California | 94609 | United States |
| Children's Hospital of Orange County- Site Number : 840074 | Orange | California | 92868 | United States |
| Stanford Unviersity MC Dept of Genetics- Site Number : 840022 | Palo Alto | California | 94305 | United States |
| UC Davis MIND Institute- Site Number : 840010 | Sacramento | California | 95817 | United States |
| University of California at San Diego- Site Number : 840007 | San Diego | California | 92093 | United States |
| University of California at San Francisco- Site Number : 840051 | San Francisco | California | United States |
| Children's Hospital Colorado - Aurora- Site Number : 840069 | Aurora | Colorado | 80045 | United States |
| University of Colorado at Denver Genetics Site Number : 840068 | Aurora | Colorado | 80045 | United States |
| Yale University School of Medicine- Site Number : 840047 | New Haven | Connecticut | 06510 | United States |
| Children's National Medical Center - Washington- Site Number : 840067 | Washington D.C. | District of Columbia | 20010 | United States |
| University of Florida Dept of Genetics- Site Number : 840083 | Gainesville | Florida | 32610 | United States |
| University Of Miami SOM- Site Number : 840006 | Miami | Florida | 33136 | United States |
| Pediatric Endocrine Associates- Site Number : 840017 | Tampa | Florida | 33612 | United States |
| Emory University School of Medicine- Human Genetics- Site Number : 840060 | Decatur | Georgia | 30033 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011 | Chicago | Illinois | 60611 | United States |
| Rush University Medical Center Genetics- Site Number : 840079 | Chicago | Illinois | 60612 | United States |
| Indianapolis University School of Medicine- Site Number : 840027 | Indianapolis | Indiana | 46202 | United States |
| University of Iowa- Site Number : 840032 | Iowa City | Iowa | 52242 | United States |
| University of Louisville- Genetics- Site Number : 840030 | Louisville | Kentucky | 40202 | United States |
| John Hopkins- Site Number : 840044 | Baltimore | Maryland | United States |
| Massachusetts General Hospital-Genetics- Site Number : 840062 | Boston | Massachusetts | 02114 | United States |
| Boston Children's Hospital - PIN- Site Number : 840093 | Boston | Massachusetts | 2115 | United States |
| Baystate Medical Center- Site Number : 840002 | Springfield | Massachusetts | 01199 | United States |
| University of Michigan Pediatrics- Site Number : 840107 | Ann Arbor | Michigan | 48109 | United States |
| University of Minnesota Medical Center Pediatrics- Site Number : 840076 | Minneapolis | Minnesota | 55455 | United States |
| Washington University- Site Number : 840100 | St Louis | Missouri | 63110 | United States |
| University of Nebraska Medical Center- Site Number : 840084 | Omaha | Nebraska | 68198 | United States |
| Children's Specialty Center - Las Vegas- Site Number : 840008 | Las Vegas | Nevada | 89109 | United States |
| Joseph M. Sanzari Children's Hospital- Site Number : 840101 | Hackensack | New Jersey | 07601 | United States |
| Saint Peter's University Hospital - New Brunswick- Site Number : 840016 | New Brunswick | New Jersey | 08901 | United States |
| Northwell Health - Site Number : 840102 | Manhasset | New York | 11030 | United States |
| New York University School Of Medicine- Site Number : 840040 | New York | New York | 10016 | United States |
| State University of New York- Site Number : 840052 | Syracuse | New York | 13210 | United States |
| Duke University Medical Center Genetics Dept- Site Number : 840037 | Durham | North Carolina | 27710 | United States |
| Cincinnati Children's Hospital-Genetics- Site Number : 840041 | Cincinnati | Ohio | United States |
| Nationwide Children's Hospital- Site Number : 840091 | Columbus | Ohio | 43205 | United States |
| Oregon Health & Science University (OHSU)- Site Number : 840080 | Portland | Oregon | 97239 | United States |
| Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089 | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of Philadelphia- Site Number : 840034 | Philadelphia | Pennsylvania | United States |
| University of Pittsburgh- Site Number : 840023 | Pittsburgh | Pennsylvania | 15224 | United States |
| Rhode Island Hospital Genetics- Site Number : 840053 | Providence | Rhode Island | 02903 | United States |
| Greenwood Genetic Center - Greenville- Site Number : 840055 | Greenville | South Carolina | 29605 | United States |
| Baylor Institute of Metabolic Disease- Site Number : 840058 | Dallas | Texas | 75226 | United States |
| University of Utah Health Hospital- Site Number : 840092 | Salt Lake City | Utah | 84132 | United States |
| University of Virginia School of Medicine-Pediatrics- Site Number : 840078 | Charlottesville | Virginia | 22908 | United States |
| Children's Hospital of The King's Daughters- Site Number : 840072 | Norfolk | Virginia | 23507 | United States |
| Seattle Children's Hospital- Site Number : 840028 | Seattle | Washington | 98105 | United States |
| University Of Washington Medical Center- Site Number : 840059 | Seattle | Washington | 98195 | United States |
| Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054 | Milwaukee | Wisconsin | 53226 | United States |
| Investigational Site Number : 153032 | Buenos Aires | 1192 | Argentina |
| Investigational Site Number : 153130 | San Luis | 5700 | Argentina |
| Investigational Site Number : 151069 | Westmead | New South Wales | 2145 | Australia |
| Investigational Site Number : 150944 | Brussels | 1090 | Belgium |
| Investigational Site Number : 151668 | Leuven | 3000 | Belgium |
| Investigational Site Number : 154008 | Roeselare | 8800 | Belgium |
| Private Practice - Dr. Fabiano Klaus- Site Number : 076001 | Porto Alegre | Rio Grande do Sul | 90035-002 | Brazil |
| Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076007 | Campinas | São Paulo | 13034-685 | Brazil |
| Instituto Fernandes Figueira - FIOCRUZ- Site Number : 076004 | Rio de Janeiro | 22250-020 | Brazil |
| Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 076002 | São Paulo | 04020-041 | Brazil |
| Investigational Site Number : 124014 | Calgary | Alberta | T2E 7Z4 | Canada |
| Investigational Site Number : 124002 | Edmonton | Alberta | T6G 2B7 | Canada |
| Investigational Site Number : 124011 | Vancouver | British Columbia | V6H 3N1 | Canada |
| Investigational Site Number : 124019 | Winnipeg | Manitoba | R3A 1R9 | Canada |
| Investigational Site Number : 124021 | London | Ontario | N6A 5A5 | Canada |
| Investigational Site Number : 124018 | Ottawa | Ontario | K1K 4L2 | Canada |
| Investigational Site Number : 124006 | Toronto | Ontario | M5G 1X8 | Canada |
| Investigational Site Number : 124001 | Sherbrooke | Quebec | J1K 2R1 | Canada |
| Investigational Site Number : 152001 | Talcahuano | Biobio | 2687000 | Chile |
| Investigational Site Number : 152003 | Los Ángeles | Chile |
| Investigational Site Number : 170001 | Bogotá | Colombia |
| Investigational Site Number : 150659 | Prague | 12808 | Czechia |
| Investigational Site Number : 151229 | Copenhagen | 2100 | Denmark |
| Investigational Site Number : 818002 | Cairo | 11231 | Egypt |
| Investigational Site Number : 818001 | Cairo | Egypt |
| Investigational Site Number : 250004 | Bordeaux | 33076 | France |
| Investigational Site Number : 250001 | Bron | 69677 | France |
| Investigational Site Number : 250002 | Marseille | 13885 | France |
| Investigational Site Number : 250003 | Paris | 75015 | France |
| Investigational Site Number : 250006 | Vandœuvre-lès-Nancy | 54511 | France |
| Investigational Site Number : 151368 | Hamburg | 20246 | Germany |
| Investigational Site Number : 150031 | Mainz | 55131 | Germany |
| Investigational Site Number : 344001 | Kowloon | Hong Kong |
| Investigational Site Number : 356003 | Ahmedabad | 380015 | India |
| Investigational Site Number : 356001 | Kolkata | 700017 | India |
| Investigational Site Number : 356002 | Vellore | 632004 | India |
| Investigational Site Number : 360001 | Jakarta | Indonesia |
| Investigational Site Number : 154161 | Dublin | D01 F772 | Ireland |
| Investigational Site Number : 380004 | Cusano Milanino | Milano | 20095 | Italy |
| Investigational Site Number : 380007 | Milan | Milano | 20142 | Italy |
| Investigational Site Number : 380006 | Rome | Roma | 00165 | Italy |
| Investigational Site Number : 380002 | Ancona | 60126 | Italy |
| Investigational Site Number : 380003 | Genova | 16147 | Italy |
| Investigational Site Number : 380005 | Naples | Italy |
| Investigational Site Number : 380008 | Padova | 35128 | Italy |
| Investigational Site Number : 392102 | Nankoku | Kochi | 783-8505 | Japan |
| Investigational Site Number : 392101 | Tokyo | 157-8535 | Japan |
| Investigational Site Number : 154225 | Kuwait City | Kuwait |
| Investigational Site Number : 4220004 | Beirut | 11072210 | Lebanon |
| Investigational Site Number : 458001 | Kuala Lumpur | 50586 | Malaysia |
| Investigational Site Number : 458002 | Kuala Lumpur | 59100 | Malaysia |
| Investigational Site Number : 151171 | Amsterdam | Netherlands |
| Investigational Site Number : 152029 | Rotterdam | 3015 CE | Netherlands |
| Investigational Site Number : 154264 | Lahore | Pakistan |
| Investigational Site Number : 154266 | Lahore | Pakistan |
| Investigational Site Number : 608001 | Manila | 1000 | Philippines |
| Investigational Site Number : 150575 | Warsaw | 04-730 | Poland |
| Investigational Site Number : 6200002 | Coimbra | 3000-075 | Portugal |
| Investigational Site Number : 154177 | Cluj-Napoca | 400006 | Romania |
| Investigational Site Number : 643001 | Moscow | 119571 | Russia |
| Investigational Site Number : 643002 | Moscow | 125412 | Russia |
| Investigational Site Number : 682001 | Al Qaţīf | Saudi Arabia |
| Investigational Site Number : 682004 | Mecca | 24246 | Saudi Arabia |
| Investigational Site Number : 703001 | Bratislava | 833 40 | Slovakia |
| Investigational Site Number : 153191 | Daejeon | South Korea |
| Investigational Site Number : 153225 | Jeonnam | South Korea |
| Investigational Site Number : 153212 | Seoul | South Korea |
| Investigational Site Number : 153222 | Seoul | South Korea |
| Investigational Site Number : 7520001 | Stockholm | 171 76 | Sweden |
| Investigational Site Number : 154093 | Chiayi City | Taiwan |
| Investigational Site Number : 152246 | Kaohsiung City | 833 | Taiwan |
| Investigational Site Number : 151500 | Putz City, Chia-yi | Taiwan |
| Investigational Site Number : 151179 | Taichung | 404 | Taiwan |
| Investigational Site Number : 152153 | Tainan | 710 | Taiwan |
| Investigational Site Number : 151182 | Taipei | 100 | Taiwan |
| Investigational Site Number : 152321 | Taipei | 104 | Taiwan |
| Investigational Site Number : 764002 | Bangkok | 10400 | Thailand |
| Investigational Site Number : 764003 | Bangkok | 10400 | Thailand |
| Investigational Site Number : 764004 | Bangkok | 10700 | Thailand |
| Investigational Site Number : 764001 | Bangkok | Thailand |
| Investigational Site Number : 154240 | Birmingham | England | B4 6NH | United Kingdom |
| Investigational Site Number : 151123 | London | England | NW3 2QG | United Kingdom |
| Investigational Site Number : 152156 | Glasgow | Glasgow City | G51 4TF | United Kingdom |
| Investigational Site Number : 151646 | London | London, City of | WC1N 3BG | United Kingdom |
| Investigational Site Number : 150303 | London | London, City of | WC1N 3JH | United Kingdom |
| Investigational Site Number : 152361 | Salford | Manchester | M6 8HD | United Kingdom |
| Investigational Site Number : 150455 | Manchester | Missouri | M13 9WL | United Kingdom |
| Investigational Site Number : 154212 | Hanoi | Vietnam |
| 24675674 | Background | Beck M, Arn P, Giugliani R, Muenzer J, Okuyama T, Taylor J, Fallet S. The natural history of MPS I: global perspectives from the MPS I Registry. Genet Med. 2014 Oct;16(10):759-65. doi: 10.1038/gim.2014.25. Epub 2014 Mar 27. |
| ID | Term |
|---|---|
| D008059 | Mucopolysaccharidosis I |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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