Disorder of GPI anchor biosynthesis
MONDO:0024321A disease that has its basis in the disruption of GPI anchor biosynthetic process.
Also known as: GPI anchor biosynthetic process disease, GPIBD, disorder of GPI anchor biosynthetic process, glycosylphosphatidylinositol biosynthesis defect
76 clinical trials for this condition and its sub-types.
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Broader categories
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PNH drug iptacopan tested in Real-World setting – but study cut short
Disease control TerminatedThis study looked at how well the drug iptacopan works for people with paroxysmal nocturnal hemoglobinuria (PNH) when used in everyday medical practice in Russia. Only 6 adults took part, and the study was terminated early. Researchers measured whether patients' hemoglobin levels…
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 12:06 UTC
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BCX9930 safety checked in PNH patients who benefited from treatment
Disease control TerminatedThis study looked at the long-term safety of BCX9930, an oral medication, for people with paroxysmal nocturnal hemoglobinuria (PNH) who had already benefited from the drug in a previous study. It included 28 participants and aimed to monitor side effects. The study was terminated…
Phase: PHASE2 • Sponsor: BioCryst Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC