Glycogen storage disease II
MONDO:0009290Glycogen storage disease due to acid maltase deficiency (AMD) is an autosomal recessive trait leading to metabolic myopathy that affects cardiac and respiratory muscles in addition to skeletal muscle and other tissues. AMD represents a wide spectrum of clinical presentations caused by an accumulation of glycogen in lysosomes: Glycogen storage disease due to acid maltase deficiency, infantile onset, non-classic infantile onset and adult onset. Early onset forms are more severe and often fatal.
Also known as: Alpha-1,4-glucosidase acid deficiency, GAA glycogen storage disease, GSD due to acid maltase deficiency, GSD type 2, GSD type II, Pompe Disease, Pompe disease, acid maltase deficiency
279 clinical trials for this condition and its sub-types.
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Broader categories
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Heart drug tested to fight COVID-19 complications
Disease control TerminatedThis study tested a drug called CardiolRx in 90 hospitalized COVID-19 patients who also had heart disease or risk factors like diabetes or high blood pressure. The goal was to see if the drug could lower the chance of death, needing intensive care, or having heart problems. The t…
Phase: PHASE2, PHASE3 • Sponsor: Cardiol Therapeutics Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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Blood filter during heart bypass may prevent organ damage, but trial stopped early
Disease control TerminatedThis study tested a device called Efferon LPS that filters blood during heart surgery when a heart-lung machine is used. The goal was to see if it could prevent multiple organ failure, especially kidney damage. Only 14 people took part before the study was stopped, so the results…
Phase: NA • Sponsor: Efferon JSC • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Pompe disease drug trial halted after just 3 patients
Disease control TerminatedThis study tested an oral drug called duvoglustat in adults with Pompe disease, a rare genetic disorder that causes muscle weakness. Only 3 people took part, and the study was stopped early. Researchers looked at safety and whether the drug could improve walking distance and othe…
Phase: PHASE2 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jun 26, 2026 16:43 UTC
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Heart surgery recovery: can patients manage their own precautions?
Symptom relief TerminatedThis study looked at whether letting patients manage their own recovery precautions after heart surgery leads to a better quality of life compared to following standard sternal precautions. Ten adults who had heart surgery were followed for up to a year. Researchers checked pain,…
Phase: NA • Sponsor: University of Chicago • Aim: Symptom relief
Last updated Jun 27, 2026 07:56 UTC
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Painkiller showdown: which is safer for the heart?
Knowledge-focused TerminatedThis study looked at how two common pain relievers—celecoxib (a Coxib) and naproxen (an NSAID)—affect blood clotting in people with rheumatoid arthritis who also have heart disease or are at high risk for it. All participants took low-dose aspirin daily. The goal was to see if on…
Phase: PHASE4 • Sponsor: Platelet and Thrombosis Research, LLC • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:32 UTC
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Scientists gather leftover surgical tissue to unlock secrets of bone disease
Knowledge-focused TerminatedThis study collected bone, cartilage, skin, and fat tissue from 50 patients undergoing surgery at Children's National Medical Center. The goal was to grow cells from these tissues in the lab and analyze them to better understand bone and connective tissue diseases. The study was …
Sponsor: National Institute of Dental and Craniofacial Research (NIDCR) • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:52 UTC