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.
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.
Pompe Disease Registry Protocol
-
University Alabama at Birmingham- Site Number : 840106, Birmingham, Alabama, United States, 35233
Barrow Neurological Institute- Site Number : 840087, Phoenix, Arizona, United States, 85013
Phoenix Children's Hospital- Site Number : 840003, Phoenix, Arizona, United States, 85013
University of Arizona- Site Number : 840015, Tucson, Arizona, United States, 85724
Arkansas Childrens Hospital- Site Number : 840109, Little Rock, Arkansas, United States, 72202
University of Arkansas for Medical Sciences- Site Number : 840113, Little Rock, Arkansas, United States, 72205
University of California at Irvine- Site Number : 840036, Irvine, California, United States, 90211
Loma Linda University- Genetics- Site Number : 840070, Loma Linda, California, United States, 92354
Southern California Permanente Medical Group- Site Number : 840108, Los Angeles, California, United States, 90027
USC Health Sciences Center Dept of Genetics- Site Number : 840082, Los Angeles, California, United States, 90033
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
For general information about clinical research, read Learn About Studies.
to
ALL
No
Genzyme, a Sanofi Company,
Study Director, STUDY_DIRECTOR, Genzyme, a Sanofi Company
2034-01-31