Pompe Disease Registry
Pompe Disease Registry
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:
Study Design Time Perspective: Retrospective and Prospective
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
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Birmingham, Alabama 35233, United States
Little Rock, Arkansas 72202, United States
Little Rock, Arkansas 72205, United States
Irvine, California 90211, United States
Loma Linda, California 92354, United States
Los Angeles, California 90027, United States
Los Angeles, California 90033, United States
Oakland, California 94609, United States
Orange, California 92868, United States
San Diego, California 92123, United States
San Francisco, California 94143, United States
Stanford, California 94305, United States
Aurora, Colorado 80045, United States
New Haven, Connecticut 06510, United States
Washington D.C., District of Columbia 20010, United States
Gainesville, Florida 32610, United States
Jacksonville, Florida 32207, United States
Decatur, Georgia 30033, United States
Aurora, Illinois 80045, United States
Chicago, Illinois 60611, United States
Chicago, Illinois 60611, United States
Chicago, Illinois 60612, United States
Indianapolis, Indiana 46202, United States
Kansas City, Kansas 66160, United States
Louisville, Kentucky 40202, United States
Portland, Maine 04102, United States
Boston, Massachusetts 02114, United States
Boston, Massachusetts 02115, United States
Springfield, Massachusetts 01199, United States
Ann Arbor, Michigan 48109, United States
Detroit, Michigan 48201, United States
Grand Rapids, Michigan 49503, United States
Grand Rapids, Michigan 49525, United States
Minneapolis, Minnesota 55404, United States
Minneapolis, Minnesota 55404, United States
Minneapolis, Minnesota 55454, United States
Minneapolis, Minnesota 55454, United States
Columbia, Missouri 65212, United States
Kansas City, Missouri 64108, United States
Omaha, Nebraska 68198, United States
Las Vegas, Nevada 89109, United States
Hackensack, New Jersey 07601, United States
New Brunswick, New Jersey 08901, United States
Paterson, New Jersey 07503, United States
Huntington, New York 11743, United States
New York, New York 10029, United States
New York, New York 10032, United States
Rochester, New York 14642, United States
Syracuse, New York 13210, United States
Charlotte, North Carolina 28232, United States
Durham, North Carolina 27710, United States
Cincinnati, Ohio 45219, United States
Cleveland, Ohio 44106, United States
Portland, Oregon 97239, United States
Portland, Oregon 97239, United States
Portland, Oregon 97239, United States
Hershey, Pennsylvania 17033, United States
Hershey, Pennsylvania 17033, United States
Philadelphia, Pennsylvania 19104, United States
Philadelphia, Pennsylvania 19104, United States
Pittsburgh, Pennsylvania 15224, United States
Pittsburgh, Pennsylvania 15224, United States
Providence, Rhode Island 02903, United States
Greenville, South Carolina 29605, United States
Nashville, Tennessee 37212, United States
Dallas, Texas 75226, United States
Fort Worth, Texas 76104, United States
Salt Lake City, Utah 84132, United States
Charlottesville, Virginia 22908, United States
Norfolk, Virginia 23507, United States
Seattle, Washington 98195, United States
Tacoma, Washington 98405, United States
Caba, Buenos Aires, Buenos Aires C1415EKV, Argentina
CABA, Buenos Aires 1424, Argentina
Fortaleza, Ceará 60150-160, Brazil
Fortaleza, Ceará 60430-270, Brazil
Cuiabá, Mato Grosso 78025-000, Brazil
Porto Alegre, Rio Grande do Sul 90035903, Brazil
Ribeirão Preto, São Paulo 14049-900, Brazil
Rio de Janeiro, 22250-020, Brazil
São Paulo, 04020-041, Brazil
São Paulo, 05403-000, Brazil
São Paulo, 05403-000, Brazil
Nyiregyháza, 4400, Hungary
Tao Yuan County, 333, Taiwan
Cardiff, Cardiff [Caerdydd Gb-crd] CF14 4XW, United Kingdom