Glycogen Storage Disease - 49 Studies Found

Not yet recruiting : Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease
: Pompe Disease
: 2014-09-11
:
  • Genetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase

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
:
  • Device: Muscle ultrasound Ultr
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 : CPAP for Infantile Pompe Disease
: Pompe Disease
: 2015-03-21
: Procedure: continuous positive airway pressure CPAP training for muscle strength
Recruiting : Immune Modulation Therapy for Pompe Disease
: Pompe Disease
: 2015-08-10
:
  • Drug: Rituximab
  • Drug: intravenous immune g
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
Recruiting : Prospective Follow-up of Patients With Glycogen Storage Disease Type III
: Neuromuscular Disorders
: 2012-03-21
:
  • Procedure: Different motor function tests

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