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Glycogen Storage Disease - 119 Studies Found
Recruiting |
: Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease : Pompe Disease : 2012-08-13 : Other: Observational This is a longitudinal study focused on the emerging natural history of Infantile P |
Recruiting |
: Pompe Disease QMUS and EIM : Pompe Disease : 2016-04-11 :
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Completed |
: Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease : Pompe Disease : 2005-06-03 |
Completed |
: Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry : Pompe Disease : 2011-07-07 : Other: Drawing blood spots from Newborns Dry blood spots would be taken for determination of Alpha Gluco |
Completed |
: Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease :
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Completed |
: CPAP for Infantile Pompe Disease : Pompe Disease : 2015-03-21 : Procedure: continuous positive airway pressure CPAP training for muscle strength |
Approved for marketing |
: Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease :
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Recruiting |
: Immune Modulation Therapy for Pompe Disease : Pompe Disease : 2015-08-10 :
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Not yet recruiting |
: Prevalence of Pompe's Disease in Respiratory Clinics : Pompe's Disease : 2015-08-17 : Other: Prospective observational study Prospective observational study collecting data during a clinic v |
Recruiting |
: A Long-term Study for the Outcome of Pompe Disease : Pompe Disease : 2015-03-21 |