MeSH D030342 - inborn genetic diseases
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Competitive Landscape
Events Timeline
Commercial
Clinical Trials
Competitive landscape
D009358:Hereditary congenital and neonatal diseases and abnormalities
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D030342:Â
Inborn genetic diseases
D000071070:Familial multiple lipomatosis
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D000072661:Ciliopathies
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D000077428:Gata2 deficiency
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D000081207:Primary immunodeficiency diseases
D000083083:Laminopathies
D000312:Congenital adrenal hyperplasia
D000699:Congenital pain insensitivity
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D000745:Hemolytic anemia congenital
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D001260:Ataxia telangiectasia
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D002636:Cherubism
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D003550:Cystic fibrosis
D003966:Camurati-engelmann syndrome
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D004392:Dwarfism
D006453:Hemoglobinopathies
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D007619:Kartagener syndrome
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D008382:Marfan syndrome
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D008661:Inborn errors metabolism
D009136:Muscular dystrophies
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D009261:Nail-patella syndrome
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D009386:Hereditary neoplastic syndromes
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D009800:Oculocerebrorenal syndrome
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D009958:Orofaciodigital syndromes
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D010004:Primary hypertrophic osteoarthropathy
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D010009:Osteochondrodysplasias
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D010013:Osteogenesis imperfecta
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D010381:Pelger-huet anomaly
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D012873:Genetic skin diseases
D014898:Werner syndrome
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D015499:Inborn errors renal tubular transport
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D015785:Hereditary eye diseases
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D017436:Kallmann syndrome
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D019896:Alpha 1-antitrypsin deficiency
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D020271:Nervous system heredodegenerative disorders
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D020294:Congenital myasthenic syndromes
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D024741:Hypertrophic cardiomyopathy familial
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D025063:Chromosome disorders
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D025861:Inherited blood coagulation disorders
D029502:Hypoplastic anemia congenital
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D031845:Hajdu-cheney syndrome
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D040181:X-linked genetic diseases
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D046589:Cadasil
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D050010:Familial dysalbuminemic hyperthyroxinemia
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D050174:Y-linked genetic diseases
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D052159:Frasier syndrome
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D053306:Hyper-igm immunodeficiency syndrome
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D053840:Brugada syndrome
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D055947:Loeys-dietz syndrome
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D056660:Hereditary autoinflammatory diseases
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D056684:Yellow nail syndrome
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D056685:Costello syndrome
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D056731:Donohue syndrome
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D056735:Autoimmune lymphoproliferative syndrome
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D058631:Pycnodysostosis
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D058747:Charge syndrome
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D065768:Lennox gastaut syndrome
D000096803:Imprinting disorders
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Events Timeline
Commercial
Company Name | Drug Name | Trade Name | Patent Expires | Approval Date | Revenue | Period |
|---|---|---|---|---|---|---|
| CSL Behring | .alpha.1-proteinase inhibitor human | Respreeza |  | 2015-08-20 |  |  |
| Regeneron Pharmaceuticals | Rilonacept | Rilonacept Regeneron (previously Arcalyst) |  | 2009-10-23 |  |  |
| Kiniksa Pharmaceuticals | Rilonacept | Arcalyst |  | 2008-02-27 |  |  |
Clinical Trials
Historical Success Rate
Phase 1
14%1/7
Phase 2
17%4/23
Phase 3
25%3/12
Approved: 1Overall Success rate: 1%
All Clinical Trials
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All
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Company name | Phase 1 | Phase 2 | Phase 3 | Phase 4 | Drug Name |
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