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. 2018 May;7(2):145–147. doi: 10.5582/irdr.2018.01056

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).