Заболявания:
Glycogen Storage Disease Type II
Pompe Disease
Спонсор: Genzyme, a Sanofi Company
Налично на:
БГ
Обобщение
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.
Описание
Study Design Time Perspective: Retrospective and Prospective
Кой може да участва
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
Места на провеждане
41
Argentina (1)
Investigational Site Number : 153195
Caba, Buenos Aires , Buenos Aires
Австралия (1)
Investigational Site Number : 036013
Westmead , New South Wales
Белгия (1)
Investigational Site Number : 150722
Antwerp
Бразилия (1)
Hospital Universitario Walter Cantidio- Site Number : 076011
Fortaleza , Ceará
COMPLETED
България (1)
Investigational Site Number : 100001
Sofia
Канада (1)
Investigational Site Number : 124014
Calgary , Alberta
Chile (1)
Investigational Site Number : 152002
Punta Arenas
COMPLETED
Китай (1)
Investigational Site Number : 152380
Beijing
TERMINATED
Colombia (1)
Investigational Site Number : 170001
Bogotá
Хърватия (1)
Investigational Site Number : 1910001
Zagreb
Чехия (1)
Investigational Site Number : 150809
Prague
Дания (1)
Investigational Site Number : 151229
Copenhagen
Франция (1)
Investigational Site Number : 250010
Bordeaux
COMPLETED
Гърция (1)
Investigational Site Number : 300001
Thessaloniki
Hong Kong (1)
Investigational Site Number : 344001
Kowloon
Унгария (1)
Investigational Site Number : 3480009
Debrecen
Индия (1)
Investigational Site Number : 356003
Ahmedabad
Израел (1)
Investigational Site Number : 3760002
Beersheba
Италия (1)
Investigational Site Number : 380020
Gussago , Brescia
Япония (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
Нидерландия (1)
Investigational Site Number : 152029
Rotterdam
Pakistan (1)
Investigational Site Number : 154264
Karachi
Philippines (1)
Investigational Site Number : 608001
City of Muntinlupa
Полша (1)
Investigational Site Number : 150575
Warsaw
Румъния (1)
Investigational Site Number : 154099
Bucharest
Russia (1)
Investigational Site Number : 643002
Moscow
COMPLETED
Saudi Arabia (1)
Investigational Site Number : 682001
Al Qaţīf
COMPLETED
Сърбия (1)
Investigational Site Number : 6880001
Belgrade
COMPLETED
Сингапур (1)
Investigational Site Number : 702001
Singapore
Словакия (1)
Investigational Site Number : 703001
Bratislava
COMPLETED
Южна Корея (1)
Investigational Site Number : 153178
Yangsan , Gyeongsangnam-do
COMPLETED
Taiwan (1)
Investigational Site Number : 151500
Chiayi City
Тайланд (1)
Investigational Site Number : 764002
Bangkok
COMPLETED
United Arab Emirates (1)
Investigational Site Number : 7840001
Dubai
Великобритания (1)
Investigational Site Number : 151340
Cambridge , Cambridgeshire
САЩ (1)
University of Alabama at Birmingham- Site Number : 840106
БГ
EN