Table 1. Clinical trials with Aurora kinase inihibitors.
No. | Diseases | No. | Diseases |
---|---|---|---|
1 | 21-Hydroxyulase Deficiency | 60 | Langerhans Cell Histiocytosis |
2 | Albinism | 61 | Laron Syndrome |
3 | Alport Syndrome | 62 | Leber Hereditary Optic Neuropathy |
4 | Amyotrophic Lateral Sclerosis | 63 | Long Chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency |
5 | Angelman Syndrome | 64 | Lymphangioleiomyomatosis (LAM) |
6 | Arginase Deficiency | 65 | Lysine Urinary Protein Intolerance |
7 | Asphyxiating Thoracic Dystrophy (Jeune Syndrome) | 66 | Lysosomal Acid Lipase Deficiency |
8 | Atypical Hemolytic Uremic Syndrome | 67 | Maple Syrup Urine Disease |
9 | Autoimmune Encephalitis | 68 | Marfan Syndrome |
10 | Autoimmune Hypophysitis | 69 | McCune-Albright Syndrome |
11 | Autoimmune Insulin Receptopathy (Type B insulin resistance) | 70 | Medium Chain Acyl-CoA Dehydrogenase Deficiency |
12 | Beta-ketothiolase Deficiency | 71 | Methylmalonic Academia |
13 | Biotinidase Deficiency | 72 | Mitochondrial Encephalomyopathy |
14 | Cardic Ion Channelopathies | 73 | Mucopolysaccharidosis |
15 | Carnitine Deficiency | 74 | Multi-Focal Motor Neurothy |
16 | Castleman Disease | 75 | Multiple Acyl-CoA Dehydrogenase Deficiency |
17 | Charcot-Marie-Tooth Disease | 76 | Multiple Sclerosis |
18 | Citrullinemia | 77 | Multiple System Atrophy |
19 | Congenital Adrenal Hypoplasia | 78 | Myotonic Dystrophy |
20 | Congenital Hyperinsulinemic Hypoglycemia | 79 | NAGS Deficiency |
21 | Congenital Myasthenic Syndrome | 80 | Neonatal Diabetes Mellitus |
22 | Congenital Myotonia Syndrome (Non-Dystrophic Myotonia, NDM) | 81 | Neuromyelitis Optica |
23 | Congenital Scoliosis | 82 | Niemann-Pick Disease |
24 | Coronary Artery Ectasia | 83 | Non-Syndromic Deafness |
25 | Diamond-Blackfan Anemia | 84 | Noonan Syndrome |
26 | Erdheim -Chester Disease | 85 | Ornithine Transcarbamylase Deficiency |
27 | Fabry Disease | 86 | Osteogenesis Imperfecta (Brittle Bone Disease) |
28 | Familial Mediterranean Fever | 87 | Parkinson Disease (Young-onset, Early-onset) |
29 | Fanconi Anemia | 88 | Paroxysmal Nocturnal Hemoglobinuia |
30 | Galactosemia | 89 | Peutz-Jeghers Syndrome |
31 | Gaucher's Disease | 90 | Phenylketonuria |
32 | General Myathenic Gravis | 91 | POEMS Syndrome |
33 | Gitelman Syndrome | 92 | Porphyria |
34 | Glutaric Acidemia Type I | 93 | Prader-Willi Syndrome |
35 | Glycogen Storage Disease (Type I, II) | 94 | Primary Combined Immune Deficiency |
36 | Hemophilia | 95 | Primary Hereditary Dystonia |
37 | Hepatolenticular Degeneration (Wilson Disease) | 96 | Primary Light Chain Amyloidosis |
38 | Hereditary Angioedema (HAE) | 97 | Progressive Familial Intrahepatic Cholestasis |
39 | Hereditary Epidermolysis Bullosa | 98 | Progressive Muscular Dystrophies |
40 | Hereditary Fructose Intolerance | 99 | Propionic Acidemia |
41 | Hereditary Hypomagnesemia | 100 | Pulmonary Alveolar Proteinosis |
42 | Hereditary Multi-infarct Dementia (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalqpathy, CADASIL) |
101 | Pulmonary Cystic Fibrosis |
102 | Retinitis Pigmentosa | ||
103 | Retinoblastoma | ||
43 | Hereditary Spastic Paraplegia | 104 | Severe Congenital Neutropenia |
44 | Holocarboxylase Synthetase Deficiency | 105 | Severe Myoclonic Epilepsy In Infaricy (Dravet Syndrome) |
45 | Homocysteinemia | 106 | Sickle Cell Disease |
46 | Homozygous Hypercholesterolemia | 107 | Silver-Russell Syndrome |
47 | Huntington Disease | 108 | Sitosterolemia |
48 | Hyperornithinaemia-Hyperammonaemia-Hhomocit rullinuria Syndrome |
109 | Spinal and Bulbar Muscular Atrophy (Kennedy Disease) |
110 | Spinal Muscular Atrophy | ||
49 | Hyperphenylalaninemia | 111 | Spinocerebellar Ataxia |
50 | Hypophosphatasia | 112 | Systemic Sclerosis |
51 | Hypophosphatemia Rickets | 113 | Tetrahydrobiopterin Deficiency |
52 | Idiopathic Cardiomyopathy | 114 | Tuberous Sclerosis Complex |
53 | Idiopathic Hypogonadotropic Hypogonadism | 115 | Tyrosinemia |
54 | Idiopathic Pulmonary Arterial Hypertension | 116 | Very Long Chain Acyl-CoA Dehydrogenase Deficiency |
55 | Idiopathic Pulmonary Fibrosis | 117 | Williams Syndrome |
56 | IgG4 related Disease | 118 | Wiskott-Aldrich Syndrome |
57 | Inborn Errors of Bile Acid Synthesis | 119 | X-linked Agammaglobulinemia |
58 | Isovaleric Acidemia | 120 | X-linked Ldrenoleuko Dystrophy |
59 | Kallmann Syndrome | 121 | X-linked Lymphoproliferative Disease |
*From the Notice on the First National List of Rare Diseases in China jointly issued by five bodies, including the National Health Commission. (1).