Pompe Disease Clinical Trials

A listing of Pompe Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 35 clinical trials
Pompe Pregnancy Sub-Registry

pregnancy outcomes, including complications and infant growth, in all women with Pompe disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with alglucosidase

alglucosidase alfa
  • 0 views
  • 23 Jan, 2022
  • 14 locations
Pompe Pregnancy Sub-Registry

pregnancy outcomes, including complications and infant growth, in all women with Pompe disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with alglucosidase

alglucosidase alfa
  • 84 views
  • 23 Jan, 2022
  • 14 locations
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease

myozyme
immunosuppressive
alglucosidase alfa
lumizyme
mutation analysis
  • 17 views
  • 01 Oct, 2021
  • 1 location
Frequency of Pompe Disease in Patients With Myalgia With or Without Hyper Ckemia - Data From the Reference Center (CERCA)

Pompe's disease is a lysosomal storage disease of autosomal recessive genetic transmission due to a deficiency in acid alpha glucosidase. This enzyme deficiency leads to glycogen overload in all

deficiency
replacement therapy
  • 0 views
  • 03 Nov, 2021
  • 1 location
Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders

, including patients with the following neuromuscular disorders: histologically confirmed inclusion body myositis (IBM), genetically confirmed late-onset Pompe disease (LOPD), genetically confirmed spinal

dyspnea
exercise tests
  • 0 views
  • 18 Jun, 2021
  • 1 location