Qualitative or quantitative protein defects in neuromuscular diseases
MONDO:001613928 clinical trials for this condition and its sub-types.
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Broader categories
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New drug aims to save hearts in duchenne muscular dystrophy
Disease control CompletedThis study tested an oral drug called ifetroban in 46 males aged 7 and older with Duchenne muscular dystrophy (DMD). The goal was to see if the drug is safe and can help prevent or treat heart problems, which are a leading cause of death in DMD. Participants received either ifetr…
Phase: PHASE2 • Sponsor: Cumberland Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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Experimental drug ataluren tested for safety in duchenne MD patients
Disease control CompletedThis study tested the safety of a drug called ataluren (also known as Translarna) in 270 people with Duchenne muscular dystrophy caused by a specific type of genetic mistake (nonsense mutation). Participants had already taken ataluren in earlier studies. The goal was to monitor s…
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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Simple frailty quiz may help predict chemo risks for seniors with stomach or bowel cancer
Knowledge-focused CompletedThis study looked at whether two simple frailty questionnaires (G8 and VES-13) can predict serious side effects from chemotherapy in people 65 and older with gastrointestinal cancers. Researchers followed 72 patients receiving standard chemotherapy and checked their frailty, nutr…
Sponsor: Ankara Etlik City Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:07 UTC
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New study tracks muscle decline in rare disease to guide future treatments
Knowledge-focused CompletedThis study followed 52 people with limb-girdle muscular dystrophy 2I (LGMD2I) for up to two years to learn more about how the disease changes over time. Researchers measured walking ability, muscle strength, heart function, and daily activities. The goal was to better understand …
Sponsor: Genethon • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:04 UTC
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Health warnings in booze app put to the test
Knowledge-focused CompletedThis study looked at whether showing health warning messages in a mobile app where people buy alcohol affects their purchasing. Over 11,700 adults in Sweden took part. One group saw rotating health warnings, while the other saw standard messages. The researchers tracked purchases…
Phase: NA • Sponsor: World Health Organization • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:05 UTC