Laminopathy
MONDO:0021106A rare genetic disorder caused by mutations in genes encoding proteins of the nuclear lamina.
25 clinical trials for this condition and its sub-types, 3 tagged with Laminopathy itself.
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Sub-types of Laminopathy
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Familial partial lipodystrophy 13 trials · 14 incl. sub-types
10 sub-types
- Familial partial lipodystrophy, Dunnigan type 4 trials
- AKT2-related familial partial lipodystrophy 0 trials
- CIDEC-related familial partial lipodystrophy 0 trials
- LIPE-related familial partial lipodystrophy 0 trials
- PLIN1-related familial partial lipodystrophy 0 trials
- PPARG-related familial partial lipodystrophy 0 trials
- Autosomal semi-dominant severe lipodystrophic laminopathy 0 trials
- Familial partial lipodystrophy, Kobberling type 0 trials
- Lipodystrophy, familial partial, type 8 0 trials
- Lipodystrophy, familial partial, type 9 0 trials
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Hutchinson-Gilford progeria syndrome 3 trials
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Autosomal dominant Emery-Dreifuss muscular dystrophy 0 trials · 3 incl. sub-types
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Greenberg dysplasia 2 trials
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Buschke-Ollendorff syndrome 0 trials
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Pelger-Huet anomaly 0 trials
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3 sub-types
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Atypical Werner syndrome 0 trials
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Restrictive dermopathy 1 0 trials
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Custom-Made genetic drug targets rare brain disorder in one patient
Disease control OngoingThis study tests a personalized medicine called an antisense oligonucleotide, designed specifically for one person with autosomal dominant leukodystrophy (ADLD), a rare genetic brain disease. The treatment aims to slow or stop the disease by targeting the underlying genetic mutat…
Phase 1/2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jul 15, 2026 00:00 UTC
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New hope for rare fat disorder: daily leptin shots target blood sugar and fats
Disease control OngoingThis Phase 3 trial tests whether a daily injection of metreleptin (a lab-made leptin hormone) can improve blood sugar and fat levels in people with partial lipodystrophy, a rare condition where fat is distributed abnormally. About 69 participants will receive either metreleptin o…
Phase 3 • Sponsor: Chiesi Farmaceutici S.p.A. • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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New drug combo aims to slow Progeria's effects
Disease control By invitation onlyThis study tests whether adding everolimus to the existing drug lonafarnib can better control progeria, a rare disease that causes rapid aging in children. About 80 children with confirmed progeria will take both pills by mouth. The first part finds the safest dose of everolimus,…
Phase 1/2 • Sponsor: Boston Children's Hospital • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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New hope for kids with rapid-aging disease: drug combo tested
Disease control OngoingThis study tests a new drug called progerinin, given together with the standard medicine lonafarnib, in 10 children with Hutchinson-Gilford progeria syndrome (a rare disease that causes rapid aging). The goal is to find the best dose and check if the combination is safe and toler…
Phase 2 • Sponsor: PRG Science & Technology Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 09:03 UTC