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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT00358943
Other study ID # DIREGC07009
Secondary ID ICGG Gaucher Reg
Status Recruiting
Phase
First received
Last updated
Start date April 1, 1991
Est. completion date January 31, 2034

Study information

Verified date April 2024
Source Sanofi
Contact Trial Transparency email recommended (Toll free number for US &
Phone 800-633-1610
Email Contact-Us@sanofi.com
Is FDA regulated No
Health authority
Study type Observational [Patient Registry]

Clinical Trial Summary

The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The objectives of the Registry are: - To enhance understanding of the variability, progression, identification, and natural history of Gaucher disease, with the ultimate goal of better guiding and assessing therapeutic intervention. - To assist the Gaucher medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. - To characterize the Gaucher disease population. - To evaluate the long-term effectiveness of imiglucerase and of eliglustat. Gaucher Pregnancy Sub-registry: The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.


Recruitment information / eligibility

Status Recruiting
Enrollment 12000
Est. completion date January 31, 2034
Est. primary completion date January 31, 2034
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility Inclusion Criteria: ICGG Gaucher Registry - All patients with a confirmed diagnosis of Gaucher disease are eligible for inclusion in the Registry. Confirmed diagnosis is defined as a documented ß-glucocerebrosidase deficiency and/or mutation in the ß-glucocerebrosidase gene. - For all patients, appropriate patient authorization will be obtained. Gaucher Pregnancy Sub-registry: - be enrolled in the ICGG Gaucher Registry. - be pregnant, or have been pregnant with appropriate medical documentation available. - provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed. Exclusion Criteria: - No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry.

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
Argentina Investigational Site Number : 032012 Buenos Aires
Argentina Investigational Site Number : 150661 Caba Buenos Aires
Argentina Investigational Site Number : 032011 Córdoba
Argentina Investigational Site Number : 154176 Corrientes
Argentina Investigational Site Number : 032003 La Plata Buenos Aires
Argentina Investigational Site Number : 151085 Sarandi Buenos Aires
Australia Investigational Site Number : 151002 Adelaide South Australia
Australia Investigational Site Number : 151011 Clayton Victoria
Australia Investigational Site Number : 151491 Douglas Queensland
Australia Investigational Site Number : 151010 Herston Queensland
Australia Investigational Site Number : 151725 Hobart Tasmania
Australia Investigational Site Number : 151015 Murdoch Western Australia
Australia Investigational Site Number : 151008 Parkville Victoria
Australia Investigational Site Number : 151069 Westmead New South Wales
Belgium Investigational Site Number : 150722 Antwerpen
Belgium Investigational Site Number : 150944 Brussel
Belgium Investigational Site Number : 152345 Brussel
Belgium Investigational Site Number : 150517 Bruxelles
Belgium Investigational Site Number : 151796 Bruxelles
Belgium Investigational Site Number : 154285 Edegem
Belgium Investigational Site Number : 151134 Gent
Belgium Investigational Site Number : 151668 Leuven
Brazil Faculdade De Medicina - Unicamp Site Number : 076005 Campinas
Brazil PUC Campinas - Sociedade Campineira de Educaçao e Instruçao Site Number : 076012 Campinas São Paulo
Brazil Hospital das Clínicas da Universidade Federal do Paraná - HCUFPR Site Number : 076010 Curitiba Paraná
Brazil Hospital De Clinicas De Porto Alegre Site Number : 076007 Porto Alegre Rio Grande Do Sul
Brazil Faculdade de Medicina de Ribeirao Preto - USP Site Number : 076001 Ribeirao Preto São Paulo
Brazil HEMORIO - Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti Site Number : 076006 Rio de Janeiro
Brazil Hospital das Clínicas da Faculdade de Medicina da USP Site Number : 076008 São Paulo
Brazil Instituto de Genética e Erros Inatos do Metabolismo - IGEIM Site Number : 076002 São Paulo
Bulgaria Investigational Site Number : 100001 Sofia
Bulgaria Investigational Site Number : 151164 Sofia
Canada Investigational Site Number : 124014 Calgary Alberta
Canada Investigational Site Number : 124002 Edmonton Alberta
Canada Investigational Site Number : 124024 Kingston Ontario
Canada Investigational Site Number : 124021 London Ontario
Canada Investigational Site Number : 124022 Moncton New Brunswick
Canada Investigational Site Number : 124001 Sherbrooke Quebec
Canada Investigational Site Number : 124006 Toronto Ontario
Canada Investigational Site Number : 124013 Toronto Ontario
Canada Investigational Site Number : 124010 Vancouver British Columbia
Canada Investigational Site Number : 124011 Vancouver British Columbia
Canada Investigational Site Number : 124019 Winnipeg Manitoba
Croatia Investigational Site Number : 1910001 Zagreb
Croatia Investigational Site Number : 1910002 Zagreb
Croatia Investigational Site Number : 1910003 Zagreb
Czechia Investigational Site Number : 150809 Praha 2
Denmark Investigational Site Number : 151229 København Ø
Dominican Republic Investigational Site Number : 2140001 Santo Domingo
Egypt Investigational Site Number : 5748 Alexandria
Egypt Investigational Site Number : 9889 Ash Sharqia
Egypt Investigational Site Number : 9888 Assuit
Egypt Investigational Site Number : 8345 Cairo
Egypt Investigational Site Number : 9829 Cairo
Egypt Investigational Site Number : 9830 Cairo
France Investigational Site Number : 250015 ANGERS Cedex 01
France Investigational Site Number : 250004 Besancon
France Investigational Site Number : 250008 Bordeaux
France Investigational Site Number : 250005 Brest
France Investigational Site Number : 250014 BRON Cedex
France Investigational Site Number : 250002 Caen
France Investigational Site Number : 250001 CLICHY Cedex
France Investigational Site Number : 250003 Garches
France Investigational Site Number : 250009 LYON Cedex 03
France Investigational Site Number : 2500013 Montpellier
France Investigational Site Number : 250006 NANTES Cedex 1
France Investigational Site Number : 250010 Paris
France Investigational Site Number : 250012 Rennes Cedex 2
France Investigational Site Number : 250018 Strasbourg
France Investigational Site Number : 250017 Toulouse
France Investigational Site Number : 250016 TOURS Cedex 9
Germany Investigational Site Number : 151686 Berlin
Germany Investigational Site Number : 150783 Düsseldorf
Germany Investigational Site Number : 154150 Gießen
Germany Investigational Site Number : 151368 Hamburg
Germany Investigational Site Number : 151369 Hamburg
Germany Investigational Site Number : 150031 Mainz
Germany Investigational Site Number : 150432 Oberhausen
Greece Investigational Site Number : 300002 Athens
Greece Investigational Site Number : 300004 Athens
Greece Investigational Site Number : 300003 Patras
Greece Investigational Site Number : 300001 Thessaloniki
Hong Kong Investigational Site Number : 344001 Kowloon
Hungary Investigational Site Number : 3480006 Budapest
Hungary Investigational Site Number : 3480003 Létavértes
Hungary Investigational Site Number : 3480007 Nyíregyháza
Hungary Investigational Site Number : 3480008 Székesfehérvár
India Investigational Site Number : 356003 Ahmedabad
India Investigational Site Number : 356004 Jaipur
India Investigational Site Number : 356001 Kolkata
India Investigational Site Number : 356005 Mumbai
India Investigational Site Number : 3560006 Secunderabad
India Investigational Site Number : 356002 Vellore
Indonesia Investigational Site Number : 360001 Jakarta
Israel Investigational Site Number : 3760003 Haifa
Israel Investigational Site Number : 3760001 Jerusalem
Israel Investigational Site Number : 3760002 Petach-Tikva
Italy Investigational Site Number : 380008 Catania
Italy Investigational Site Number : 380009 Firenze
Italy Investigational Site Number : 380006 Genova
Italy Investigational Site Number : 380004 Milano
Italy Investigational Site Number : 380005 Modena
Italy Investigational Site Number : 380011 Monza
Italy Investigational Site Number : 380002 Monza (MB)
Italy Investigational Site Number : 380010 Napoli
Italy Investigational Site Number : 380003 Padova
Italy Investigational Site Number : 380007 Roma
Italy Investigational Site Number : 380001 Udine
Japan Investigational Site Number : 153205 Abeno-ku Osaka
Japan Investigational Site Number : 153217 Chiba
Japan Investigational Site Number : 153204 Setagaya Tokyo
Japan Investigational Site Number : 153203 Yonago-shi Tottori
Jordan Investigational Site Number : 4000001 Amman
Korea, Republic of Investigational Site Number : 153191 Daejeon
Korea, Republic of Investigational Site Number : 153225 Jeonnam
Korea, Republic of Investigational Site Number : 151149 Seoul
Korea, Republic of Investigational Site Number : 153180 Seoul
Korea, Republic of Investigational Site Number : 153192 Seoul
Korea, Republic of Investigational Site Number : 153196 Seoul
Korea, Republic of Investigational Site Number : 153212 Seoul
Korea, Republic of Investigational Site Number : 153222 Seoul
Korea, Republic of Investigational Site Number : 154148 Yangsan Gyeongsangnam-do
Korea, Republic of Investigational Site Number : 153178 Yangsan-si Gyeongsangnam-do
Kuwait Investigational Site Number : 154225 Kuwait
Lebanon Investigational Site Number : 4220004 Beirut
Lebanon Investigational Site Number : 422001 Beirut
Lebanon Investigational Site Number : 422006 Douris
Lebanon Investigational Site Number : 4220002 Hazmieh
Lithuania Investigational Site Number : 154237 Vilnius
Malaysia Investigational Site Number : 458001 Jalan Pahang
Malaysia Investigational Site Number : 458002 Kuala Lumpur
Netherlands Investigational Site Number : 150149 Amsterdam
Pakistan Investigational Site Number : 154264 Karachi
Pakistan Investigational Site Number : 154266 Lahore
Peru Investigational Site Number : 604001 Arequipa
Peru Investigational Site Number : 604002 Callao
Philippines Investigational Site Number : 608001 Manila
Poland Investigational Site Number : 150575 Warszawa
Portugal Investigational Site Number : 620003 Coimbra
Portugal Investigational Site Number : 620004 Lisboa
Portugal Investigational Site Number : 620009 Ponta Delgada
Romania Investigational Site Number : 151188 Cluj Napoca
Romania Investigational Site Number : 642002 Cluj Napoca
Romania Investigational Site Number : 642001 Cluj-Napoca
Russian Federation Investigational Site Number : 643001 Moscow
Russian Federation Investigational Site Number : 643002 sait-Petersburg
Saudi Arabia Investigational Site Number : 682002 Al Mubarraz
Saudi Arabia Investigational Site Number : 682003 Jeddah
Saudi Arabia Investigational Site Number : 682004 Makkah
Serbia Investigational Site Number : 688001 Belgrade
Serbia Investigational Site Number : 688002 Belgrade
Serbia Investigational Site Number : 688003 Belgrade
Serbia Investigational Site Number : 688005 Belgrade
Serbia Investigational Site Number : 688004 Nis
Singapore Investigational Site Number : 702001 Singapore
Slovakia Investigational Site Number : 703001 Bratislava
South Africa Investigational Site Number : 154138 Johannesburg
South Africa Investigational Site Number : 710-003 Johannesburg
South Africa Investigational Site Number : 150346 Sandton
Spain Investigational Site Number : 153237 Barcelona
Spain Investigational Site Number : 154147 Barcelona
Spain Investigational Site Number : 154146 La Coruña
Spain Investigational Site Number : 151524 Madrid
Spain Investigational Site Number : 154119 Madrid
Spain Investigational Site Number : 154160 Madrid
Spain Investigational Site Number : 154163 Madrid
Spain Investigational Site Number : 154199 Madrid
Spain Investigational Site Number : 154236 Madrid
Spain Investigational Site Number : 154145 Sabadell
Spain Investigational Site Number : 154222 Santiago de Compostela
Spain Investigational Site Number : 153215 Sevilla
Spain Investigational Site Number : 153219 Sevilla
Spain Investigational Site Number : 154205 Valencia
Spain Investigational Site Number : 153213 Vigo
Spain Investigational Site Number : 153216 Zaragoza
Spain Investigational Site Number : 154238 Zaragoza
Sweden Investigational Site Number : 154095 Borås
Sweden Investigational Site Number : 151726 Malmö
Sweden Investigational Site Number : 154092 Norrköping
Switzerland Investigational Site Number : 157045 Bern
Taiwan Investigational Site Number : 154093 Chiayi
Taiwan Investigational Site Number : 152152 Hualien
Taiwan Investigational Site Number : 152244 Kaohsiung
Taiwan Investigational Site Number : 152246 Kaohsiung Hsien,
Taiwan Investigational Site Number : 151534 New Taipei City
Taiwan Investigational Site Number : 151500 Putz City, Chia-yi
Taiwan Investigational Site Number : 151181 Taichung
Taiwan Investigational Site Number : 151179 Taichung City
Taiwan Investigational Site Number : 152153 Tainan
Taiwan Investigational Site Number : 153248 Tainan
Taiwan Investigational Site Number : 151182 Taipei
Taiwan Investigational Site Number : 152226 Taipei
Taiwan Investigational Site Number : 152321 Taipei
Taiwan Investigational Site Number : 153158 Tao Yuan County
Thailand Investigational Site Number : 764001 Bangkok
Thailand Investigational Site Number : 764003 Bangkok
Thailand Investigational Site Number : 764004 Bangkok
Thailand Investigational Site Number : 764005 Khon Kaen
Turkey Investigational Site Number : 792006 Adana
Turkey Investigational Site Number : 792001 Ankara
Turkey Investigational Site Number : 792002 Ankara
Turkey Investigational Site Number : 792005 Ankara
Turkey Investigational Site Number : 792004 Istanbul
Turkey Investigational Site Number : 792007 Izmir
Turkey Investigational Site Number : 792003 Malatya
United Arab Emirates Investigational Site Number : 7840001 Dubai
United Kingdom Investigational Site Number : 152287 Birmingham
United Kingdom Investigational Site Number : 154240 Birmingham
United Kingdom Investigational Site Number : 151340 Cambridge Cambridgeshire
United Kingdom Investigational Site Number : 150303 London
United Kingdom Investigational Site Number : 151123 London
United Kingdom Investigational Site Number : 151646 London
United Kingdom Investigational Site Number : 150455 Manchester
United Kingdom Investigational Site Number : 152361 Salford
United States University of Michigan Pediatrics Site Number : 840107 Ann Arbor Michigan
United States University of Colorado at Denver Genetics Site Number : 840068 Aurora Colorado
United States John Hopkins Site Number : 840044 Baltimore Maryland
United States Tower Hematology/Oncology Medical Group Site Number : 840056 Beverly Hills California
United States Boston Children's Hospital Site Number : 840092 Boston Massachusetts
United States Massachusetts General Hospital-Genetics Site Number : 840062 Boston Massachusetts
United States Carolinas Medical Center Hospital Site Number : 840065 Charlotte North Carolina
United States University of Virginia School of Medicine-Pediatrics Site Number : 840078 Charlottesville Virginia
United States Ann and Robert Lurie Children's Hospital Site Number : 840011 Chicago Illinois
United States Ann and Robert Lurie Children's Hospital Site Number : 840013 Chicago Illinois
United States Rush University Medical Center Genetics Site Number : 840079 Chicago Illinois
United States Cleveland Clinic Foundation Pediatrics Site Number : 840048 Cleveland Ohio
United States UHCMC Site Number : 840119 Cleveland Ohio
United States Nationwide Children's Hospital Site Number : 840091 Columbus Ohio
United States University of Missouri Health System Department of Genetics Site Number : 840031 Columibia Missouri
United States University Hematology Oncology Site Number : 840075 Coral Springs Florida
United States Baylor Research Center Site Number : 840058 Dallas Texas
United States Emory University School of Medicine- Human Genetics Site Number : 840060 Decatur Georgia
United States Children's Hospital of Michigan Site Number : 840066 Detroit Michigan
United States LSD Data Registry Site LLC Site Number : 840094 Dublin Ohio
United States Duke University Medical Center Genetics Dept Site Number : 840037 Durham North Carolina
United States O&O Alpan, LLC Site Number : 840025 Fairfax Virginia
United States Cook Children's Health Care System Site Number : 840024 Fort Worth Texas
United States Infusion Associates Site Number : 840050 Grand Rapids Michigan
United States Greenwood Genetcs Site Number : 840055 Greenville South Carolina
United States Joseph M. Sanzari Children's Hospital Site Number : 840101 Hackensack New Jersey
United States Indianapolis University School of Medicine Site Number : 840027 Indianapolis Indiana
United States University of Iowa Site Number : 840032 Iowa City Iowa
United States University of California at Irvine Site Number : 840036 Irvine California
United States The Atwal Clinic Site Number : 840112 Jacksonville Florida
United States University of Florida Pediatrics Genetics Site Number : 840121 Jacksonville Florida
United States Children's Mercy Hospital- Pediatrics Site Number : 840063 Kansas City Missouri
United States Children's Specialty Center of Nevada Site Number : 840008 Las Vegas Nevada
United States Arkansas Childrens Hospital Site Number : 840109 Little Rock Arkansas
United States University of Arkansas for Medical Sciences Site Number : 840113 Little Rock Arkansas
United States Southern California Permanente Medical Group Site Number : 840108 Los Angeles California
United States USC Health Sciences Center Dept of Genetics Site Number : 840082 Los Angeles California
United States University of Louisville- Genetics Site Number : 840030 Louisville Kentucky
United States Northwell Health Site Number : 840102 Manhasset New York
United States University Of Miami SOM Site Number : 840006 Miami Florida
United States Children's Hospital of Wisconsin-Pediatrics Site Number : 840054 Milwaukee Wisconsin
United States Children's Health Care Site Number : 840114 Minneapolis Minnesota
United States Children's Hospital and Clinics of Minnesota Site Number : 840046 Minneapolis Minnesota
United States University of Minnesota Medical Center Pediatrics Site Number : 840076 Minneapolis Minnesota
United States Atlantic Health System Site Number : 840099 Morristown New Jersey
United States Vanderbilt University Hospital-Pediatrics Site Number : 840049 Nashville Tennessee
United States St. Peter's University Hospital Site Number : 840016 New Brunswick New Jersey
United States Yale Site Number : 840047 New Haven Connecticut
United States Ochsner Medical Center Site Number : 840120 New Orleans Louisiana
United States Tulane University Medical Center Site Number : 840001 New Orleans Louisiana
United States Metropolitan Hospital Center Site Number : 840110 New York New York
United States Mt. Sinai School of Medicine Site Number : 840005 New York New York
United States New York University School Of Medicine Site Number : 840040 New York New York
United States Site Number : 840026 New York New York
United States Children's Hospital of the Kings Daughters Site Number : 840072 Norfolk Virginia
United States University of Nebraska Medical Center- Pediatrics Site Number : 840084 Omaha Nebraska
United States Children's Hospital of Orange County Site Number : 840074 Orange California
United States Stanford Unviersity MC Dept of Genetics Site Number : 840022 Palo Alto California
United States St. Joseph's Children's Hospital Site Number : 840057 Paterson New Jersey
United States Children's Hospital of Philadelphia HUP Medical Genetics Site Number : 840089 Philadelphia Pennsylvania
United States Children's Hospital of Philadelphia Site Number : 840034 Philadelphia Pennsylvania
United States Phoenix Children's Hospital Site Number : 840003 Phoenix Arizona
United States University of Pittsburgh Site Number : 840023 Pittsburgh Pennsylvania
United States Maine Medical Center Pediatrics Site Number : 840064 Portland Maine
United States Oregon Health & Science University Site Number : 840080 Portland Oregon
United States Rhode Island Hospital Genetics Site Number : 840053 Providence Rhode Island
United States University of Rochester Medical Center SOM Site Number : 840105 Rochester New York
United States Kaiser Permanente Hospital Site Number : 840042 Sacramento California
United States UC Davis MIND Institute Site Number : 840010 Sacramento California
United States Washington University of St. Louis Site Number : 840100 Saint Louis Missouri
United States University of Utah Department of Medical Genetics Site Number : 840043 Salt Lake City Utah
United States Loma Linda University Children's Hospital Site Number : 840117 San Bernardino California
United States University of California at San Diego Site Number : 840007 San Diego California
United States University of California at San Francisco Site Number : 840051 San Francisco California
United States Seattle Children's Hospital Site Number : 840028 Seattle Washington
United States University Of Washington Medical Center Site Number : 840059 Seattle Washington
United States State University of New York Site Number : 840052 Syracuse New York
United States Multicare Health System ¿ Mary Bridge Childrens Hospital Site Number : 840115 Tacoma Washington
United States Pediatric Endocrine Associates Site Number : 840017 Tampa Florida
United States University of Florida-Genetics Site Number : 840096 Tampa Florida
United States University of Arizona Site Number : 840015 Tucson Arizona
United States New York Medical College Site Number : 840039 Valhalla New York
United States Children's National Medical Center Site Number : 840067 Washington District of Columbia
Vietnam Investigational Site Number : 154212 Hanoi
Vietnam Investigational Site Number : 704002 Ho Chi Minh City

Sponsors (1)

Lead Sponsor Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

United States,  Vietnam,  Argentina,  Australia,  Belgium,  Brazil,  Bulgaria,  Canada,  Croatia,  Czechia,  Denmark,  Dominican Republic,  Egypt,  France,  Germany,  Greece,  Hong Kong,  Hungary,  India,  Indonesia,  Israel,  Italy,  Japan,  Jordan,  Korea, Republic of,  Kuwait,  Lebanon,  Lithuania,  Malaysia,  Netherlands,  Pakistan,  Peru,  Philippines,  Poland,  Portugal,  Romania,  Russian Federation,  Saudi Arabia,  Serbia,  Singapore,  Slovakia,  South Africa,  Spain,  Sweden,  Switzerland,  Taiwan,  Thailand,  Turkey,  United Arab Emirates,  United Kingdom, 

References & Publications (3)

El-Beshlawy A, Tylki-Szymanska A, Vellodi A, Belmatoug N, Grabowski GA, Kolodny EH, Batista JL, Cox GF, Mistry PK. Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry. Mol Genet Metab. 2017 Jan-Feb;120(1-2):47-56. doi: 10.1016/j.ymgme.2016.12.001. Epub 2016 Dec 6. — View Citation

Mistry PK, Batista JL, Andersson HC, Balwani M, Burrow TA, Charrow J, Kaplan P, Khan A, Kishnani PS, Kolodny EH, Rosenbloom B, Scott CR, Weinreb N. Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry. Am J Hematol. 2017 Sep;92(9):929-939. doi: 10.1002/ajh.24801. Epub 2017 Jul 7. — View Citation

Weinreb NJ, Goldblatt J, Villalobos J, Charrow J, Cole JA, Kerstenetzky M, vom Dahl S, Hollak C. Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment. J Inherit Metab Dis. 2013 May;36(3):543-53. doi: 10.1007/s10545-012-9528-4. Epub 2012 Sep 14. Erratum In: J Inherit Metab Dis. 2014 Jan;37(1):147. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary ICGG Gaucher Registry: To provide the Gaucher medical community with recommendations for monitoring patients and to provide reports on patient outcomes to help optimize patient care 42 years
Primary Gaucher Pregnancy Sub-registry: To track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with imiglucerase 42 years
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