Disorder of glycosylation
MONDO:0024322A disease that has its basis in the disruption of glycosylation.
Also known as: disorder of glycosylation, glycosylation disease
74 clinical trials for this condition and its sub-types, 0 tagged with Disorder of glycosylation itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Disorder of glycosylation
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Disorder of GPI anchor biosynthesis 0 trials · 73 incl. sub-types
11 sub-types
- Paroxysmal nocturnal hemoglobinuria 73 trials Sub-types →
- 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 Sub-types →
- Neurodevelopmental disorder with hypotonia and cerebellar atrophy, with or without seizures 0 trials
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DHDDS-CDG 1 trial
1 sub-type
- Retinitis pigmentosa 59 0 trials
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Congenital myasthenic syndrome 12 0 trials
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Schneckenbecken dysplasia 0 trials
Most studied deeper sub-types
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New complement inhibitor takes on established PNH drug in phase III trial
Disease control Not yet recruitingResearchers are testing an experimental drug called VSA012 against eculizumab in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have not received complement inhibitor therapy. The trial enrolls about 70 participants and compares how well each treatment controls hemolys…
Phase 3 • Sponsor: Bisirna Therapeutics (Suzhou) Co., Ltd. • Aim: Disease control
Last updated Sep 13, 2026 00:00 UTC
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Can a new injection tame the blood cell destruction of PNH?
Disease control Not yet recruitingThis Phase II trial tests an experimental drug called SGB-9768 in adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder where red blood cells break apart too early. The study gives the drug by injection under the skin and measures how well it lowers a marke…
Phase 2 • Sponsor: Suzhou Sanegene Bio Inc. • Aim: Disease control
Last updated Sep 06, 2026 00:00 UTC
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Can an oral pill tame a rare blood disease?
Disease control Not yet recruitingThis study tests whether an experimental oral drug called lanoracopan hydrochloride can safely and effectively treat paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that causes red blood cells to break apart. The trial enrolls adults aged 18 to 75 with PNH, inclu…
Sponsor: Peking Union Medical College Hospital • Aim: Disease control
Last updated Aug 28, 2026 00:00 UTC
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A pill that could replace infusions for a rare blood disease?
Disease control Not yet recruitingThis trial compares an experimental oral drug, FXS6837, to the standard infusion eculizumab in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that causes red blood cells to break apart. About 90 adults who have not previously used complement inhibito…
Phase 3 • Sponsor: Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. • Aim: Disease control
Last updated Aug 07, 2026 00:00 UTC
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New hope for PNH: experimental drug aims to outdo soliris
Disease control Not yet recruitingThis study tests a new drug called NM8074 in 12 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already taking Soliris. PNH is a rare blood disorder where the immune system destroys red blood cells, causing anemia and fatigue. NM8074 is designed to block a different…
Phase 2 • Sponsor: NovelMed Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:06 UTC
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New drug SLN12140 aims to control PNH in early trial
Disease control Not yet recruitingThis phase II trial tests the drug SLN12140 in 10 adults with PNH who have never taken complement inhibitors. The goal is to see if SLN12140 can reduce red blood cell destruction (measured by LDH levels) and improve hemoglobin. Participants receive different doses over 12 to 64 w…
Phase 2 • Sponsor: Linno Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:01 UTC
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New hope for PNH patients: LP-005 shows promise in long-term trial
Disease control Not yet recruitingThis study looks at the long-term safety and effectiveness of an injection called LP-005 in adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It is for people who already completed an earlier LP-005 study. The goal is to see if LP-005 can help control …
Phase 2 • Sponsor: Longbio Pharma • Aim: Disease control
Last updated Jun 27, 2026 08:02 UTC
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New pill could replace infusions for rare blood disorder
Disease control Not yet recruitingThis Phase 3 trial tests NTQ5082, an oral capsule, against the standard infusion eculizumab for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 78 adults who have not had complement inhibitor therapy will be enrolled. The main goal is to see if NTQ5082 can…
Phase 3 • Sponsor: Nanjing Chia-tai Tianqing Pharmaceutical • Aim: Disease control
Last updated Jun 26, 2026 17:59 UTC
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New pill shows promise for rare blood disorder in Long-Term trial
Disease control Not yet recruitingThis Phase 3 trial tests NTQ5082 capsules, a new drug that blocks a protein called CFB, in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study will follow 78 adults who previously benefited from NTQ5082 or took ano…
Phase 3 • Sponsor: Nanjing Chia-tai Tianqing Pharmaceutical • Aim: Disease control
Last updated Jun 26, 2026 17:40 UTC