GENETIC DISEASES
Clinical trials for GENETIC DISEASES explained in plain language.
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Hope for duchenne: new drug targets rare gene mutation in phase 3 trial
Disease control Recruiting nowThis study tests an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). About 70 boys aged 7 to 16 who can still walk will receive either the drug or a placebo. The main goal is to see if the dru…
Matched conditions: GENETIC DISEASES
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC
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Scientists seek genetic clues to mysterious fever diseases
Knowledge-focused Recruiting nowThis study aims to learn more about the genetics and natural history of autoinflammatory diseases, which cause repeated fevers and inflammation. Researchers will study up to 5,000 people, including patients, their relatives, and healthy volunteers. Participants provide blood and …
Matched conditions: GENETIC DISEASES
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Aug 15, 2026 00:00 UTC
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Gene hunt in children could unlock secrets of rare metabolic diseases
Knowledge-focused Recruiting nowThis study looks at children with suspected or confirmed genetic and metabolic disorders to find new disease-causing gene mutations. Researchers will analyze blood samples for DNA and metabolites, and in some cases take a small skin sample. The goal is to better understand these …
Matched conditions: GENETIC DISEASES
Sponsor: University of Texas Southwestern Medical Center • Aim: Knowledge-focused
Last updated Jul 04, 2026 00:00 UTC
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Researchers to track rare heart disease in 36 patients
Knowledge-focused Recruiting nowThis study aims to describe how a genetic heart condition called PKP2-ACM naturally progresses over time in people who receive standard care. Researchers will monitor heart rhythm, biomarkers, and quality of life in 36 participants aged 12 and older. The goal is to better underst…
Matched conditions: GENETIC DISEASES
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:08 UTC
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Major study launches to unravel rare genetic diseases affecting immunity and the brain
Knowledge-focused Recruiting nowThis study aims to learn more about rare genetic diseases that affect both the immune system and the nervous system. Researchers will collect medical information and samples from 1,000 participants, including patients, their relatives, and healthy volunteers. The goal is to bette…
Matched conditions: GENETIC DISEASES
Sponsor: Imagine Institute • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:05 UTC
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Scientists launch national study to track immune weakness linked to severe COVID
Knowledge-focused Recruiting nowThis study follows 500 adults in France who have either a genetic defect or autoantibodies that weaken their type I interferon immune response. Researchers will collect blood samples and health data over 1 to 4 years to track infections, autoimmune diseases, and cancers. The goal…
Matched conditions: GENETIC DISEASES
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jun 26, 2026 15:22 UTC