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Disease ReportsInborn genetic diseases
MeSH D030342 - inborn genetic diseases
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Commercial
Clinical Trials
Competitive landscape
D009358:Hereditary congenital and neonatal diseases and abnormalities
0 Companies
0 Drugs
Success rate
D030342: 
Inborn genetic diseases
$
Success rate
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
$
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D000083083:Laminopathies
$
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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
$
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D006453:Hemoglobinopathies
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D007619:Kartagener syndrome
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D008382:Marfan syndrome
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D008661:Inborn errors metabolism
$
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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
$
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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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D016738:Alagille syndrome
$
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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
$
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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
$
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D000096803:Imprinting disorders
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Events Timeline
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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 PharmaceuticalsRilonacept Rilonacept Regeneron (previously Arcalyst)  2009-10-23   
Kiniksa PharmaceuticalsRilonacept 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
Therapeutic Areas:
All
Classification:
All
Approval:
All
Status:
All
Phases:
All
Company name
Phase 1
Phase 2
Phase 3
Phase 4
Drug Name
Agios Pharmaceuticals
Regeneron Pharmaceuticals
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