Conditions:
Glycogen Storage Disease Type II
Pompe Disease
Sponsor: Genzyme, a Sanofi Company
trial.available_in:
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Overview
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes.
The objectives of the Registry are:
* To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention.
* To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care.
* To characterize the Pompe disease population.
* To evaluate the long-term effectiveness of alglucosidase alfa.
Description
Study Design Time Perspective: Retrospective and Prospective
Who can participate
Inclusion Criteria:
All patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations.
Exclusion Criteria:
There are no exclusion criteria in this Registry
Locations
41
Argentina (1)
Investigational Site Number : 153195
Caba, Buenos Aires , Buenos Aires
Australia (1)
Investigational Site Number : 036013
Westmead , New South Wales
Belgium (1)
Investigational Site Number : 150722
Antwerp
Brazil (1)
Hospital Universitario Walter Cantidio- Site Number : 076011
Fortaleza , Ceará
COMPLETED
Bulgaria (1)
Investigational Site Number : 100001
Sofia
Canada (1)
Investigational Site Number : 124014
Calgary , Alberta
Chile (1)
Investigational Site Number : 152002
Punta Arenas
COMPLETED
China (1)
Investigational Site Number : 152380
Beijing
TERMINATED
Colombia (1)
Investigational Site Number : 170001
Bogotá
Croatia (1)
Investigational Site Number : 1910001
Zagreb
Czech Republic (1)
Investigational Site Number : 150809
Prague
Denmark (1)
Investigational Site Number : 151229
Copenhagen
France (1)
Investigational Site Number : 250010
Bordeaux
COMPLETED
Greece (1)
Investigational Site Number : 300001
Thessaloniki
Hong Kong (1)
Investigational Site Number : 344001
Kowloon
Hungary (1)
Investigational Site Number : 3480009
Debrecen
India (1)
Investigational Site Number : 356003
Ahmedabad
Israel (1)
Investigational Site Number : 3760002
Beersheba
Italy (1)
Investigational Site Number : 380020
Gussago , Brescia
Japan (1)
Investigational Site Number : 392105
Ginowan , Okinawa
COMPLETED
Jordan (1)
Investigational Site Number : 4000001
Amman
Kuwait (1)
Investigational Site Number : 154225
Kuwait City
COMPLETED
Lebanon (1)
Investigational Site Number : 4220004
Beirut
Malaysia (1)
Investigational Site Number : 458001
Kuala Lumpur
Netherlands (1)
Investigational Site Number : 152029
Rotterdam
Pakistan (1)
Investigational Site Number : 154264
Karachi
Philippines (1)
Investigational Site Number : 608001
City of Muntinlupa
Poland (1)
Investigational Site Number : 150575
Warsaw
Romania (1)
Investigational Site Number : 154099
Bucharest
Russia (1)
Investigational Site Number : 643002
Moscow
COMPLETED
Saudi Arabia (1)
Investigational Site Number : 682001
Al Qaţīf
COMPLETED
Serbia (1)
Investigational Site Number : 6880001
Belgrade
COMPLETED
Singapore (1)
Investigational Site Number : 702001
Singapore
Slovakia (1)
Investigational Site Number : 703001
Bratislava
COMPLETED
South Korea (1)
Investigational Site Number : 153178
Yangsan , Gyeongsangnam-do
COMPLETED
Taiwan (1)
Investigational Site Number : 151500
Chiayi City
Thailand (1)
Investigational Site Number : 764002
Bangkok
COMPLETED
United Arab Emirates (1)
Investigational Site Number : 7840001
Dubai
United Kingdom (1)
Investigational Site Number : 151340
Cambridge , Cambridgeshire
United States (1)
University of Alabama at Birmingham- Site Number : 840106
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