Glycogen storage disease due to acid maltase deficiency, late-onset
MONDO:0018485Glycogen storage disease due to acid maltase deficiency, late onset (AMDL), a form of Glycogen storage disease due to acid maltase deficiency (AMD), a degenerative metabolic myopathy particularly affecting respiratory and skeletal muscles, is characterized by an accumulation of glycogen in lysosomes.
Also known as: Alpha-1,4-glucosidase acid deficiency, late onset, Alpha-1,4-glucosidase acid deficiency, late-onset, GSD due to acid maltase deficiency, late onset, GSD due to acid maltase deficiency, late-onset, GSD type 2, late onset, GSD type 2, late-onset, GSD type II, late onset, GSD type II, late-onset
28 clinical trials for this condition and its sub-types, 19 tagged with Glycogen storage disease due to acid maltase deficiency, late-onset itself.
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New pompe disease drug moves to first human tests
Disease control CompletedThis early-phase study tested a new drug called ABX1100 in 46 people, including healthy volunteers and patients with late-onset Pompe disease. The main goal was to check safety and how the drug moves through the body. Researchers gave single or multiple doses to find the right am…
Early phase 1 • Sponsor: Aro Biotherapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:30 UTC
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Pompe disease drug combo shows Long-Term safety in phase 3 trial
Disease control CompletedThis study tested the long-term safety of a combination therapy (ATB200 and AT2221) in 119 adults with late-onset Pompe disease who had completed a prior study. Participants received the drugs for an extended period, and researchers tracked side effects, walking distance, and lun…
Phase 3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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New drug cocktail aims to boost walking and breathing in pompe patients
Disease control CompletedThis phase 3 trial tested a new treatment for adults with late-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. Participants received either the experimental combo (cipaglucosidase alfa plus miglustat) or the current standard therapy (alglucosidase…
Phase 3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jun 26, 2026 16:41 UTC
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Home breathing training shows promise for pompe patients
Symptom relief CompletedThis study tested whether high-dose inspiratory muscle training (IMT) using a handheld device is safe and feasible for people with late-onset Pompe disease. 34 adults with stable Pompe disease did remote breathing exercises. Researchers measured changes in respiratory strength an…
Sponsor: Duke University • Aim: Symptom relief
Last updated Jun 27, 2026 07:53 UTC
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Pompe disease may raise risk of brain aneurysms, new study investigates
Knowledge-focused CompletedThis study investigates whether people with late-onset Pompe disease have a higher risk of severe blood vessel problems in the brain and aorta. Researchers will collect medical and genetic data from adults with Pompe disease and from those who have had a brain aneurysm or bleedin…
Sponsor: Hospitales Universitarios Virgen del Rocío • Aim: Knowledge-focused
Last updated Jul 08, 2026 00:00 UTC