Disorder of GPI anchor biosynthesis

MONDO:0024321

A 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

73 clinical trials for this condition and its sub-types, 0 tagged with Disorder of GPI anchor biosynthesis itself.

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Where it sits in the disease tree

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Part of

↑ Metabolic disease (7369) ↑ Disorder of glycosylation (74)

Sub-types of Disorder of GPI anchor biosynthesis

Paroxysmal nocturnal hemoglobinuria 73 trials
2 sub-types
- Paroxysmal nocturnal hemoglobinuria 1 0 trials
- Paroxysmal nocturnal hemoglobinuria 2 0 trials
Developmental and epileptic encephalopathy, 55 0 trials
Developmental and epileptic encephalopathy, 77 0 trials
Developmental and epileptic encephalopathy, 80 0 trials
Glycosylphosphatidylinositol biosynthesis defect 15 0 trials
Glycosylphosphatidylinositol biosynthesis defect 16 0 trials
Glycosylphosphatidylinositol biosynthesis defect 17 0 trials
Glycosylphosphatidylinositol biosynthesis defect 18 0 trials
Glycosylphosphatidylinositol biosynthesis defect 25 0 trials
Inborn disorder of glycosphingolipid and glycosylphosphatidylinositol anchor glycosylation 0 trials
8 sub-types
- CHIME syndrome 0 trials
- GM3 synthase deficiency 0 trials
- Hypercoagulability syndrome due to glycosylphosphatidylinositol deficiency 0 trials
- Hyperphosphatasia-intellectual disability syndrome 0 trials Sub-types →
- Intellectual disability, autosomal recessive 53 0 trials
- Multiple congenital anomalies-hypotonia-seizures syndrome 1 0 trials
- Multiple congenital anomalies-hypotonia-seizures syndrome 2 0 trials
- Multiple congenital anomalies-hypotonia-seizures syndrome 3 0 trials
Neurodevelopmental disorder with hypotonia and cerebellar atrophy, with or without seizures 0 trials

Most studied deeper sub-types

Hyperphosphatasia with intellectual disability syndrome 1 (0) Hyperphosphatasia with intellectual disability syndrome 2 (0) Hyperphosphatasia with intellectual disability syndrome 3 (0) Hyperphosphatasia with intellectual disability syndrome 4 (0) Hyperphosphatasia with intellectual disability syndrome 5 (0) Hyperphosphatasia with intellectual disability syndrome 6 (0)

Including sub-types (73) Tagged with Disorder of GPI anchor biosynthesis (0)

Trials to join now! 23 Not yet recruiting 9 Not yet finished but already full! 22 Completed 17 Terminated 2

Disorder of GPI anchor biosynthesis

Where it sits in the disease tree

Most studied deeper sub-types

PNH drug iptacopan tested in Real-World setting – but study cut short

BCX9930 safety checked in PNH patients who benefited from treatment