Fabry Disease Registry Protocol

Status: Recruiting
Location: See all (275) locations...
Study Type: Observational
SUMMARY

The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry 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 primary objectives of the Registry are: * To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; * To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; * To characterize and describe the Fabry population as a whole; * To evaluate the long-term safety and effectiveness of Fabrazyme® Fabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated. Data from the Sub-registry are also used to fulfill various global regulatory requirements, to support product development/reimbursement, and for other research and non-research-related purposes. 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.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Fabry Registry: All patients with a confirmed diagnosis of Fabry disease who have signed the informed consent and patient authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as a documented deficiency in plasma or leukocyte αGAL (alpha-galactosidase) enzyme activity and/or mutation(s) in the gene coding for αGAL.

• Fabry Pregnancy Sub-registry:

⁃ Eligible women must:

∙ be enrolled in the Fabry 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.

Locations
United States
Alabama
University of Alabama Birmingham- Nephrology- Site Number : 840018
COMPLETED
Birmingham
University of Alabama Birmingham- Nephrology- Site Number : 840073
RECRUITING
Birmingham
Arkansas
Arkansas Childrens Hospital- Site Number : 840109
RECRUITING
Little Rock
University of Arkansas for Medical Sciences- Site Number : 840113
RECRUITING
Little Rock
Arizona
Phoenix Children's Hospital- Site Number : 840003
RECRUITING
Phoenix
University of Arizona- Site Number : 840015
RECRUITING
Tucson
California
University of California at Irvine- Site Number : 840036
RECRUITING
Irvine
Southern California Permanente Medical Group- Site Number : 840108
RECRUITING
Los Angeles
UCLA School Of Medicine- Site Number : 840088
RECRUITING
Los Angeles
USC Health Sciences Center Dept of Genetics- Site Number : 840082
COMPLETED
Los Angeles
Children's Hospital Oakland- Site Number : 840029
COMPLETED
Oakland
Children's Hospital of Orange County- Site Number : 840074
RECRUITING
Orange
Stanford Unviersity MC Dept of Genetics- Site Number : 840022
RECRUITING
Palo Alto
Kaiser Permanente Hospital- Site Number : 840042
COMPLETED
Sacramento
UC Davis MIND Institute- Site Number : 840010
RECRUITING
Sacramento
University of California at San Diego- Site Number : 840007
COMPLETED
San Diego
University of California at San Francisco- Site Number : 840051
RECRUITING
San Francisco
Connecticut
Yale - Site Number : 840047
RECRUITING
New Haven
Washington, D.c.
Children's National Medical Center- Site Number : 840067
RECRUITING
Washington D.c.
Florida
University Hematology Oncology- Site Number : 840075
RECRUITING
Coral Springs
University of Florida Dept of Genetics- Site Number : 840083
RECRUITING
Gainesville
University of Florida Pediatrics Genetics- Site Number : 840121
RECRUITING
Jacksonville
University Of Miami SOM- Site Number : 840006
RECRUITING
Miami
University of Florida-Genetics- Site Number : 840096
RECRUITING
Tampa
Georgia
Emory University School of Medicine- Human Genetics- Site Number : 840060
RECRUITING
Decatur
Iowa
University of Iowa- Site Number : 840032
RECRUITING
Iowa City
Illinois
University of Colorado at Denver Genetics- Site Number : 840068
RECRUITING
Aurora
Ann and Robert Lurie Children's Hospital- Site Number : 840013
RECRUITING
Chicago
Rush University Medical Center Genetics- Site Number : 840079
RECRUITING
Chicago
Indiana
Indianapolis University School of Medicine- Site Number : 840027
RECRUITING
Indianapolis
Kansas
University of Kansas Medical Center- Site Number : 840071
RECRUITING
Kansas City
Kentucky
University of Louisville- Genetics- Site Number : 840030
COMPLETED
Louisville
Louisiana
Ochsner Medical Center- Site Number : 840120
RECRUITING
Baton Rouge
Tulane University Medical Center- Site Number : 840001
COMPLETED
New Orleans
Massachusetts
Boston Children's Hospital- Site Number : 840092
RECRUITING
Boston
Brigham and Women's Hospital-Neurology- Site Number : 840103
RECRUITING
Boston
Massachusetts General Hospital-Genetics- Site Number : 840062
RECRUITING
Boston
Baystate Health- Site Number : 840002
RECRUITING
Springfield
Maryland
John Hopkins- Site Number : 840044
COMPLETED
Baltimore
Maine
Maine Medical Center Pediatrics- Site Number : 840064
RECRUITING
Portland
Michigan
University of Michigan Pediatrics- Site Number : 840107
RECRUITING
Ann Arbor
Children's Hospital of Michigan- Site Number : 840066
RECRUITING
Detroit
Infusion Associates- Site Number : 840050
RECRUITING
Grand Rapids
Spectrum for Health- Site Number : 840019
COMPLETED
Grand Rapids
Minnesota
Children's Health Care- Site Number : 840114
RECRUITING
Minneapolis
Children's Hospital and Clinics of Minnesota- Site Number : 840046
COMPLETED
Minneapolis
University of Minnesota Medical Center Pediatrics- Site Number : 840076
RECRUITING
Minneapolis
Missouri
University of Missouri Health System Department of Genetics- Site Number : 840031
RECRUITING
Columibia
Washington University of St. Louis- Site Number : 840100
RECRUITING
St Louis
Montana
Shodair Children's Hospital- Site Number : 840090
COMPLETED
Helena
North Carolina
Carolinas Medical Center- Site Number : 840065
RECRUITING
Charlotte
Duke University Medical Center Genetics Dept- Site Number : 840045
RECRUITING
Durham
Nebraska
University of Nebraska Medical Center- Pediatrics- Site Number : 840084
RECRUITING
Omaha
New Jersey
Cooper Health Center- Site Number : 840098
TERMINATED
Camden
Joseph M. Sanzari Children's Hospital- Site Number : 840101
RECRUITING
Hackensack
Atlantic Health System- Site Number : 840099
RECRUITING
Morristown
St. Peter's University Hospital- Site Number : 840016
RECRUITING
New Brunswick
St. Joseph's Children's Hospital- Site Number : 840057
RECRUITING
Paterson
Nevada
Children's Specialty Center of Nevada- Site Number : 840008
RECRUITING
Las Vegas
New York
Northwell Health- Site Number : 840102
RECRUITING
Manhasset
Icahn School of Medicine at Mount Sinai- Site Number : 840080
RECRUITING
New York
Metropolitan Hospital Center- Site Number : 840110
RECRUITING
New York
New York University School Of Medicine- Site Number : 840040
RECRUITING
New York
University of Rochester Medical Center SOM- Site Number : 840105
COMPLETED
Rochester
State University of New York- Site Number : 840052
RECRUITING
Syracuse
New York Medical College- Site Number : 840039
RECRUITING
Valhalla
Ohio
Cincinnati Children's Hospital-Genetics- Site Number : 840033
RECRUITING
Cincinnati
Cleveland Clinic Foundation Pediatrics- Site Number : 840048
RECRUITING
Cleveland
University Hospitals Cleveland Medical Center- Site Number : 840119
RECRUITING
Cleveland
Nationwide Children's Hospital- Site Number : 840091
RECRUITING
Columbus
Ohio State University Department of Genetics- Site Number : 840097
ACTIVE_NOT_RECRUITING
Columbus
LSD Data Registry Site LLC- Site Number : 840094
RECRUITING
Dublin
Oregon
Oregon Health and Science University- Site Number : 840116
RECRUITING
Portland
Oregon Health and Scinece University- Site Number : 840038
TERMINATED
Portland
Pennsylvania
Geisinger- Site Number : 840111
RECRUITING
Danville
Penn State University Medical Center-Neurology- Site Number : 840104
RECRUITING
Hershey
Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089
RECRUITING
Philadelphia
Children's Hospital of Philadelphia- Site Number : 840034
RECRUITING
Philadelphia
University of Pittsburgh- Site Number : 840023
RECRUITING
Pittsburgh
Rhode Island
Rhode Island Hospital Genetics- Site Number : 840053
COMPLETED
Providence
South Carolina
Greenwood Genetcs- Site Number : 840055
RECRUITING
Greenville
Tennessee
Vanderbilt University Hospital-Pediatrics- Site Number : 840049
RECRUITING
Nashville
Texas
Baylor Research Center- Site Number : 840058
RECRUITING
Dallas
Cook Children's Health Care System- Site Number : 840024
RECRUITING
Fort Worth
Utah
University of Utah Department of Medical Genetics- Site Number : 840043
RECRUITING
Salt Lake City
Virginia
University of Virginia School of Medicine-Pediatrics- Site Number : 840078
COMPLETED
Charlottesville
O&O Alpan, LLC- Site Number : 840025
RECRUITING
Fairfax
Children's Hospital of the Kings Daughters- Site Number : 840072
COMPLETED
Norfolk
Washington
Seattle Children's Hospital- Site Number : 840028
RECRUITING
Seattle
University Of Washington Medical Center- Site Number : 840059
RECRUITING
Seattle
Multicare Health System ¿ Mary Bridge Childrens Hospital- Site Number : 840115
RECRUITING
Tacoma
Wisconsin
Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054
RECRUITING
Milwaukee
Other Locations
Argentina
Investigational Site Number : 153130
COMPLETED
Buenos Aires
Investigational Site Number : 153172
COMPLETED
Buenos Aires
Investigational Site Number : 153194
RECRUITING
Catamarca
Investigational Site Number : 153032
COMPLETED
Ciudad Autónoma De Buenos Aires
Investigational Site Number : 032005
COMPLETED
La Rioja
Investigational Site Number : 032004
RECRUITING
Santa Fe
Australia
Investigational Site Number : 151002
RECRUITING
Adelaide
Investigational Site Number : 153063
RECRUITING
Brisbane
Investigational Site Number : 153040
RECRUITING
Parkville
Investigational Site Number : 153090
RECRUITING
Perth
Investigational Site Number : 036013
RECRUITING
Westmead
Investigational Site Number : 151069
RECRUITING
Westmead
Belgium
Investigational Site Number : 150722
RECRUITING
Antwerp
Investigational Site Number : 153133
RECRUITING
Antwerp
Investigational Site Number : 0560008
RECRUITING
Brussels
Investigational Site Number : 150517
RECRUITING
Brussels
Investigational Site Number : 153001
RECRUITING
Brussels
Investigational Site Number : 151668
RECRUITING
Leuven
Investigational Site Number : 154275
RECRUITING
Liège
Investigational Site Number : 153100
RECRUITING
Seraing
Brazil
Medicordis- Site Number : 076009
ACTIVE_NOT_RECRUITING
Brasília
Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076013
ACTIVE_NOT_RECRUITING
Campinas
Hospital Geral e Maternidade de Cuiabá- Site Number : 076006
ACTIVE_NOT_RECRUITING
Cuiabá
Instituto do Rim do Paraná- Site Number : 076004
ACTIVE_NOT_RECRUITING
Curitiba
Clínica Senhor do Bonfim LTDA- Site Number : 076008
ACTIVE_NOT_RECRUITING
Feira De Santana
Hospital Universitário Professor Polydoro Ernani de São Thiago - UFSC- Site Number : 076012
ACTIVE_NOT_RECRUITING
Florianópolis
Serviço de Terapia Renal de Ourinhos- Site Number : 076011
ACTIVE_NOT_RECRUITING
Ourinhos
Instituto de Medicina Integral Professor Fernando Figueira - IMIP - Site Number : 076005
ACTIVE_NOT_RECRUITING
Recife
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 076001
ACTIVE_NOT_RECRUITING
São Paulo
Saúde Center Clínica - Clínica Médica Nossa Senhora de Fátima - Site Number : 076002
ACTIVE_NOT_RECRUITING
Tapejara
CTDR - Centro de Tratamento de Doenças Renais EIRELI- Site Number : 076007
ACTIVE_NOT_RECRUITING
Uberlândia
Bulgaria
Investigational Site Number : 100001
ACTIVE_NOT_RECRUITING
Sofia
Investigational Site Number : 153169
COMPLETED
Sofia
Canada
Investigational Site Number : 124014
ACTIVE_NOT_RECRUITING
Calgary
Investigational Site Number : 124002
RECRUITING
Edmonton
Investigational Site Number : 124008
RECRUITING
Halifax
Investigational Site Number : 124005
TERMINATED
Kingston
Investigational Site Number : 124021
RECRUITING
London
Investigational Site Number : 124022
ACTIVE_NOT_RECRUITING
Moncton
Investigational Site Number : 124013
ACTIVE_NOT_RECRUITING
Montreal
Investigational Site Number : 124001
ACTIVE_NOT_RECRUITING
Sherbrooke
Investigational Site Number : 124023
RECRUITING
St. John's
Investigational Site Number : 124006
ACTIVE_NOT_RECRUITING
Toronto
Investigational Site Number : 124009
ACTIVE_NOT_RECRUITING
Toronto
Investigational Site Number : 124010
ACTIVE_NOT_RECRUITING
Vancouver
Investigational Site Number : 124011
COMPLETED
Vancouver
Investigational Site Number : 124019
RECRUITING
Winnipeg
Chile
Investigational Site Number : 152004
ACTIVE_NOT_RECRUITING
Antofagasta
Investigational Site Number : 152001
ACTIVE_NOT_RECRUITING
Coquimbo
Investigational Site Number : 152003
COMPLETED
Iquique
Investigational Site Number : 152002
TERMINATED
Santiago
Investigational Site Number : 152005
ACTIVE_NOT_RECRUITING
Vallenar
China
Investigational Site Number : 153165
TERMINATED
Beijing
Investigational Site Number : 1560003
ACTIVE_NOT_RECRUITING
Hangzhou
Investigational Site Number : 1560002
RECRUITING
Jinan
Investigational Site Number : 1560004
RECRUITING
Nanjing
Investigational Site Number : 153163
TERMINATED
Shanghai
Investigational Site Number : 1560001
RECRUITING
Shanghai
Colombia
Investigational Site Number : 170001
ACTIVE_NOT_RECRUITING
Bogotá
Croatia
Investigational Site Number : 1910001
COMPLETED
Rijena
Investigational Site Number : 1910002
RECRUITING
Zagreb
Denmark
Investigational Site Number : 153041
COMPLETED
Copenhagen
Investigational Site Number : 151229
COMPLETED
København Ø
Estonia
Investigational Site Number : 153106
ACTIVE_NOT_RECRUITING
Tartu
Finland
Investigational Site Number : 153024
ACTIVE_NOT_RECRUITING
Turku
France
Investigational Site Number : 250005
ACTIVE_NOT_RECRUITING
Bordeaux
Investigational Site Number : 250002
ACTIVE_NOT_RECRUITING
Bron
Investigational Site Number : 250001
ACTIVE_NOT_RECRUITING
Garches
Investigational Site Number : 250007
ACTIVE_NOT_RECRUITING
Lille
Investigational Site Number : 250004
ACTIVE_NOT_RECRUITING
Saint-priest-en-jarez
Investigational Site Number : 250009
RECRUITING
Strasbourg
Germany
Investigational Site Number : 153013
ACTIVE_NOT_RECRUITING
Berlin
Investigational Site Number : 154150
RECRUITING
Giessen
Investigational Site Number : 153147
RECRUITING
Münster
Investigational Site Number : 153011
ACTIVE_NOT_RECRUITING
Würzburg
Hong Kong Special Administrative Region
Investigational Site Number : 344001
RECRUITING
Kowloon
Hungary
Investigational Site Number : 3480002
RECRUITING
Budapest
Investigational Site Number : 3480003
RECRUITING
Létavértes
Investigational Site Number : 3480005
RECRUITING
Pécs
Investigational Site Number : 3480004
RECRUITING
Sopron
India
Investigational Site Number : 356003
RECRUITING
Ahmedabad
Investigational Site Number : 356001
RECRUITING
Kolkata
Investigational Site Number : 3560004
RECRUITING
Secunderabad
Investigational Site Number : 356002
RECRUITING
Vellore
Indonesia
Investigational Site Number : 360001
RECRUITING
Jakarta
Italy
Investigational Site Number : 380002
RECRUITING
Bassano Del Grappa (vi)
Investigational Site Number : 380019
RECRUITING
Bologna
Investigational Site Number : 380004
RECRUITING
Florence
Investigational Site Number : 380013
RECRUITING
Florence
Investigational Site Number : 380005
RECRUITING
Genova
Investigational Site Number : 380006
COMPLETED
Milan
Investigational Site Number : 380009
COMPLETED
Milan
Investigational Site Number : 380003
RECRUITING
Modena
Investigational Site Number : 380010
RECRUITING
Monza (mb)
Investigational Site Number : 380008
RECRUITING
Napoli
Investigational Site Number : 380007
COMPLETED
Rimini
Investigational Site Number : 380012
RECRUITING
Roma
Investigational Site Number : 380011
RECRUITING
Torino
Investigational Site Number : 380001
RECRUITING
Udine
Investigational Site Number : 380014
RECRUITING
Vittoria (rg)
Japan
Investigational Site Number : 153205
COMPLETED
Abeno-ku
Investigational Site Number : 153217
RECRUITING
Chiba
Investigational Site Number : 392007
ACTIVE_NOT_RECRUITING
Kobe
Investigational Site Number : 153201
RECRUITING
Nangoku-shi
Investigational Site Number : 392006
RECRUITING
Niigata
Investigational Site Number : 153204
RECRUITING
Tokyo
Lithuania
Investigational Site Number : 154244
RECRUITING
Vilnius
Investigational Site Number : 154652
RECRUITING
Vilnius
Malaysia
Investigational Site Number : 458001
RECRUITING
Jalan Pahang
Investigational Site Number : 458002
RECRUITING
Kuala Lumpur
Netherlands
Investigational Site Number : 151171
TERMINATED
Amsterdam
Investigational Site Number : 153004
TERMINATED
Amsterdam
Norway
Investigational Site Number : 153141
RECRUITING
Bergen
Peru
Investigational Site Number : 604002
RECRUITING
Callao
Philippines
Investigational Site Number : 608001
RECRUITING
Manila
Poland
Investigational Site Number : 150575
RECRUITING
Warsaw
Portugal
Investigational Site Number : 620005
RECRUITING
Lisbon
Investigational Site Number : 620001
RECRUITING
Porto
Republic of Korea
Investigational Site Number : 154246
RECRUITING
Busan
Investigational Site Number : 153191
RECRUITING
Daejeon
Investigational Site Number : 153228
TERMINATED
Jeju City
Investigational Site Number : 151149
RECRUITING
Seoul
Investigational Site Number : 153180
RECRUITING
Seoul
Investigational Site Number : 153188
RECRUITING
Seoul
Investigational Site Number : 153192
COMPLETED
Seoul
Investigational Site Number : 153196
RECRUITING
Seoul
Investigational Site Number : 153212
RECRUITING
Seoul
Investigational Site Number : 153222
RECRUITING
Seoul
Investigational Site Number : 154273
RECRUITING
Suwon
Investigational Site Number : 153178
COMPLETED
Yangsan
Investigational Site Number : 154208
TERMINATED
Yangsan
Romania
Investigational Site Number : 154099
RECRUITING
Bucharest
Investigational Site Number : 154164
RECRUITING
Bucharest
Investigational Site Number : 153081
COMPLETED
Cluj-napoca
Russian Federation
Investigational Site Number : 643001
RECRUITING
Moscow
Investigational Site Number : 643003
RECRUITING
Nizhny Novgorod
Investigational Site Number : 643002
RECRUITING
Saint-peterburg
Saudi Arabia
Investigational Site Number : 682002
RECRUITING
Al Mubarraz
Serbia
Investigational Site Number : 6880001
RECRUITING
Novi Sad
Singapore
Investigational Site Number : 702001
RECRUITING
Singapore
Slovakia
Investigational Site Number : 703001
COMPLETED
Bratislava
Slovenia
Investigational Site Number : 705001
RECRUITING
Slovenj Gradec
Spain
Investigational Site Number : 154146
COMPLETED
A Coruña
Investigational Site Number : 154159
COMPLETED
A Coruña
Investigational Site Number : 154135
COMPLETED
Barakaldo
Investigational Site Number : 153067
RECRUITING
Barcelona
Investigational Site Number : 153077
RECRUITING
Barcelona
Investigational Site Number : 153237
RECRUITING
Barcelona
Investigational Site Number : 154123
RECRUITING
Barcelona
Investigational Site Number : 154147
RECRUITING
Barcelona
Investigational Site Number : 154204
RECRUITING
Barcelona
Investigational Site Number : 154136
RECRUITING
Tarragona
Investigational Site Number : 153213
RECRUITING
Vigo
Sweden
Investigational Site Number : 153050
RECRUITING
Gothenburg
Investigational Site Number : 151484
RECRUITING
Stockholm
Taiwan
Investigational Site Number : 154093
RECRUITING
Chiayi City
Investigational Site Number : 152152
RECRUITING
Hualien City
Investigational Site Number : 152244
COMPLETED
Kaohsiung City
Investigational Site Number : 152246
RECRUITING
Kaohsiung City
Investigational Site Number : 153088
COMPLETED
Kaohsiung City
Investigational Site Number : 151534
RECRUITING
New Taipei City
Investigational Site Number : 151500
RECRUITING
Putz City, Chia-yi
Investigational Site Number : 151179
RECRUITING
Taichung
Investigational Site Number : 151181
RECRUITING
Taichung
Investigational Site Number : 152153
RECRUITING
Tainan City
Investigational Site Number : 153248
RECRUITING
Tainan City
Investigational Site Number : 151182
RECRUITING
Taipei
Investigational Site Number : 152226
RECRUITING
Taipei
Investigational Site Number : 152321
RECRUITING
Taipei
Investigational Site Number : 153158
RECRUITING
Taoyuan
Thailand
Investigational Site Number : 764001
RECRUITING
Bangkok
Investigational Site Number : 764003
RECRUITING
Bangkok
Investigational Site Number : 764004
RECRUITING
Bangkok
Investigational Site Number : 764005
RECRUITING
Khon Kaen
United Kingdom
Investigational Site Number : 154196
RECRUITING
Belfast
Investigational Site Number : 152287
RECRUITING
Birmingham
Investigational Site Number : 151340
RECRUITING
Cambridge
Investigational Site Number : 150303
RECRUITING
London
Investigational Site Number : 151123
RECRUITING
London
Investigational Site Number : 151646
RECRUITING
London
Investigational Site Number : 150455
RECRUITING
Manchester
Investigational Site Number : 152361
RECRUITING
Salford
Viet Nam
Investigational Site Number : 154212
RECRUITING
Hanoi
Contact Information
Primary
Trial Transparency
contact-us@sanofi.com
800-633-1610
Backup
Fabry Registry HelpLine
617-591-5500
Time Frame
Start Date: 2001-07-31
Estimated Completion Date: 2034-01-31
Participants
Target number of participants: 9000
Treatments
Patients with Fabry disease
No experimental intervention is given. A patient with Fabry Disease will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.
Pregnant women with confirmed diagnosis of Fabry
No experimental intervention is given. Pregnant women with confirmed diagnosis of Fabry that are participating in the Fabry Registry and consented to participate in the Fabry Sub-registry, regardless of whether she is receiving disease-specific therapy (such as ERT with agalsidase beta) and irrespective of the commercial product with which she may be treated.
Related Therapeutic Areas
Sponsors
Leads: Genzyme, a Sanofi Company

This content was sourced from clinicaltrials.gov