RARE DISEASES
Clinical trials for RARE DISEASES explained in plain language.
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One-shot gene editor aims to correct a brain disorder at its source
⭐️ CURE ⭐️ TerminatedThis trial tests whether a single injection of a gene-editing tool can safely correct a specific mutation in the CHD3 gene that causes Snijders Blok-Campeau syndrome, a condition marked by developmental delay and intellectual disability. The gene editor is delivered directly into…
Matched conditions: RARE DISEASES
Phase: EARLY_PHASE1 • Sponsor: Yongguo Yu • Aim: ⭐️ CURE ⭐️
Last updated Aug 01, 2026 00:00 UTC
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Russian NMOSD patients under the microscope: no new drug, just Real-World data
Knowledge-focused TerminatedThis study is not testing a new treatment. Instead, it observes how 100 adults with a rare autoimmune disease called NMOSD are managed in everyday Russian clinics. Researchers will collect medical records and follow patients over time to understand their symptoms, treatments, and…
Matched conditions: RARE DISEASES
Sponsor: AstraZeneca • Aim: Knowledge-focused
Last updated Jul 23, 2026 00:00 UTC