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90 studies found for:    Pompe
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Rank Status Study
21 Completed A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
Conditions: Pompe Disease;   Glycogen Storage Disease Type II
Intervention: Other: Observation
22 Completed Safety and Efficacy of Clenbuterol on Motor Function in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
Condition: Pompe Disease
Interventions: Drug: Clenbuterol;   Drug: Placebo
23 Recruiting A Pilot Study of Pyridostigmine in Pompe Disease
Condition: Pompe Disease
Intervention: Drug: Pyridostigmine Bromide
24 Recruiting Pompe Lactation Sub-Registry
Conditions: Glycogen Storage Disease;   Pompe Disease
Intervention: Biological: alglucosidase alfa
25 Completed Albuterol in Individuals With Late Onset Pompe Disease (LOPD)
Condition: Pompe Disease
Intervention: Drug: Albuterol
26 Not yet recruiting Search for Serum/Plasma Biomarkers in Pompe's Disease
Conditions: Late Onset Pompe Disease;   Pompe Disease
Intervention: Drug: Enzyme Replacement Agent
27 Recruiting Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Condition: Pompe Disease
Interventions: Other: Thoracic MRI;   Other: Spirometry;   Other: Inspiratory Load Compensation;   Other: Maximal Inspiratory Pressure;   Other: Resting Breathing Pattern;   Other: Respiratory Muscle Endurance Test
28 Completed Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy
Condition: Pompe Disease
Interventions: Drug: Albuterol;   Drug: Placebo
29 Recruiting Muscle Response to Enzyme Replacement Therapy in Pompe Disease
Condition: Pompe Disease
Intervention:
30 Terminated A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
Condition: Late-onset Pompe Patients Untreated or Treated With rhGAA
Intervention:
31 Recruiting Alglucosidase Alfa Pompe Safety Sub-Registry
Condition: Pompe Disease
Intervention: Biological: alglucosidase alfa
32 Unknown  Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia
Condition: Pompe Disease
Intervention:
33 Completed Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Condition: Pompe Disease
Interventions: Drug: rAAV1-CMV-GAA (study agent) Administration;   Other: RMST
34 Recruiting Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.
Conditions: Pompe Disease;   Muscle Weakness
Intervention: Other: Resistance Exercise Training
35 Recruiting VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease
Condition: Pompe Disease
Interventions: Drug: VAL-1221 3 mg/kg;   Drug: VAL-1221 10 mg/kg;   Drug: VAL-1221 30 mg/kg;   Drug: RhGAA
36 Not yet recruiting Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease
Condition: Pompe Disease
Interventions: Genetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase;   Drug: Rapamycin;   Other: Lactated Ringer's;   Drug: Rituxan;   Drug: Diphenhydramine;   Drug: Acetaminophen;   Drug: Lidocaine
37 Recruiting A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
Conditions: Pompe Disease;   Hypersensitivity Reaction
Intervention: Drug: Zavesca® Prescription
38 Unknown  CPAP for Infantile Pompe Disease
Condition: Pompe Disease
Intervention: Procedure: continuous positive airway pressure
39 Recruiting A Long-term Study for the Outcome of Pompe Disease
Condition: Pompe Disease
Intervention:
40 Unknown  Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry
Condition: Pompe Disease
Intervention: Other: Drawing blood spots from Newborns

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Study has passed its completion date and status has not been verified in more than two years.