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86 studies found for:    Pompe
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Rank Status Study
1 Recruiting A Natural History Study of Adult Onset Pompe Disease Using Muscle MRI
Condition: Pompe Disease
2 Completed Newborn Screening Assay of Pompe's Disease
Condition: Pompe Disease
Intervention: Other: Pompe disease newborn screening
3 Recruiting Pompe Pregnancy Sub-Registry
Conditions: Glycogen Storage Disease Type II (GSD-II);   Pompe Disease (Late-onset);   Glycogenesis 2 Acid Maltase Deficiency
Interventions: Biological: alglucosidase alpha;   Other: No Treatment
4 Recruiting Pompe Disease Registry
Conditions: Glycogen Storage Disease Type II;   Pompe Disease
5 Recruiting Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Condition: Pompe Disease
Intervention: Other: Observational
6 Completed Pompe Prevalence Study in Patients With Muscle Weakness Without Diagnosis
Condition: Pompe's Disease
Intervention: Procedure: blood test
7 Completed Pregnancy and Birth Outcome in Women With Pompe Disease
Condition: Pompe Disease
8 Recruiting Pompe Lactation Sub-Registry
Conditions: Glycogen Storage Disease;   Pompe Disease
Intervention: Biological: alglucosidase alfa
9 Completed The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease
Condition: Pompe Disease
Intervention: Other: respiratory muscle strength training
10 Recruiting Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle Weakness
Condition: Pompe Disease
Intervention: Other: Blood sample
11 Completed Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
Condition: Pompe Disease
12 Recruiting Biomarker for Pompe Disease
Conditions: Lysosomal Storage Diseases;   Pompe Disease
13 Recruiting Response to Diaphragmatic Pacing in Subjects With Pompe Disease
Condition: Pompe Disease
Interventions: Other: Pulmonary function testing;   Other: Resting breathing pattern;   Other: Respiratory muscle endurance test;   Other: Phrenic nerve stimulation;   Other: Maximal inspiratory pressure;   Other: Forced expiratory testing;   Other: EMG
14 Recruiting Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
Condition: Pompe Disease
Interventions: Drug: Rituximab;   Drug: Miglustat
15 Not yet recruiting Prevalence of Pompe's Disease in Respiratory Clinics
Condition: Pompe's Disease
Intervention: Other: Prospective observational study
16 Recruiting Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.
Conditions: Pompe Disease;   Muscle Weakness
Intervention: Other: Resistance Exercise Training
17 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
18 Completed Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth
Condition: Pompe's Disease
Intervention: Other: observational (no intervention)
19 Recruiting Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices
Condition: Pompe Disease
20 Terminated Study to Evaluate the Safety of AT2220 in Pompe Disease
Condition: Pompe Disease
Intervention: Drug: AT2220

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