DDrare: Database of Drug Development for Rare Diseases

Cyclosporine (gengraf ) (DrugBank: Cyclosporine)

43 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
11	重症筋無力症	0
19	ライソゾーム病	0
20	副腎白質ジストロフィー	0
26	HTLV-1関連脊髄症	0
28	全身性アミロイドーシス	0
36	表皮水疱症	0
38	スティーヴンス・ジョンソン症候群	0
39	中毒性表皮壊死症	0
40	高安動脈炎	0
41	巨細胞性動脈炎	0
42	結節性多発動脈炎	0
45	好酸球性多発血管炎性肉芽腫症	0
46	悪性関節リウマチ	0
49	全身性エリテマトーデス	0
50	皮膚筋炎／多発性筋炎	0
53	シェーグレン症候群	0
55	再発性多発軟骨炎	0
56	ベーチェット病	0
58	肥大型心筋症	0
60	再生不良性貧血	1
61	自己免疫性溶血性貧血	0
62	発作性夜間ヘモグロビン尿症	0
63	特発性血小板減少性紫斑病	0
64	血栓性血小板減少性紫斑病	0
65	原発性免疫不全症候群	0
90	網膜色素変性症	0
93	原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］	0
95	自己免疫性肝炎	0
97	潰瘍性大腸炎	0
113	筋ジストロフィー	0
162	類天疱瘡（後天性表皮水疱症を含む。）	0
164	眼皮膚白皮症	0
222	一次性ネフローゼ症候群	0
226	間質性膀胱炎（ハンナ型）	0
228	閉塞性細気管支炎	0
234	ペルオキシソーム病（副腎白質ジストロフィーを除く。）	0
269	化膿性無菌性関節炎・壊疽性膿皮症・アクネ症候群	0
274	骨形成不全症	0
283	後天性赤芽球癆	0
284	ダイアモンド・ブラックファン貧血	0
285	ファンコニ貧血	0
302	レーベル遺伝性視神経症	0
326	大理石骨病	0

11. 重症筋無力症　［臨床試験数：226，薬物数：172（DrugBank：45），標的遺伝子数：43，標的パスウェイ数：118］

Searched query = "Myasthenia", "MG"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 226 trial found

19. ライソゾーム病　［臨床試験数：784，薬物数：673（DrugBank：101），標的遺伝子数：68，標的パスウェイ数：184］

Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 784 trial found

20. 副腎白質ジストロフィー　［臨床試験数：49，薬物数：86（DrugBank：29），標的遺伝子数：18，標的パスウェイ数：112］

Searched query = "Adrenoleukodystrophy", "Adrenomyeloneuropathy", "AMN"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 49 trial found

26. HTLV-1関連脊髄症　［臨床試験数：26，薬物数：46（DrugBank：27），標的遺伝子数：37，標的パスウェイ数：123］

Searched query = "HTLV-1-associated myelopathy", "Tropical spastic paraparesis", "HTLV-1", "HTLV-I-associated myelopathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 26 trial found

28. 全身性アミロイドーシス　［臨床試験数：212，薬物数：234（DrugBank：72），標的遺伝子数：54，標的パスウェイ数：170］

Searched query = "Systemic amyloidosis", "AL amyloidosis", "Immunoglobulin light chain amyloidosis", "Amyloid light-chain amyloidosis", "Familial amyloidosis", "Familial amyloid polyneuropathy", "Senile transthyretin amyloidosis", "Senile TTR amyloidosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 212 trial found

36. 表皮水疱症　［臨床試験数：147，薬物数：170（DrugBank：40），標的遺伝子数：32，標的パスウェイ数：113］

Searched query = "Epidermolysis bullosa", "EBS", "JEB", "DDEB", "RDEB", "Kindler syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 147 trial found

38. スティーヴンス・ジョンソン症候群　［臨床試験数：11，薬物数：19（DrugBank：8），標的遺伝子数：14，標的パスウェイ数：98］

Searched query = "Stevens-Johnson syndrome", "SJS", "Mucocutaneous ocular syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 11 trial found

39. 中毒性表皮壊死症　［臨床試験数：11，薬物数：18（DrugBank：8），標的遺伝子数：11，標的パスウェイ数：101］

Searched query = "Toxic epidermal necrolysis", "Toxic epidermal necrosis", "TEN"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 11 trial found

40. 高安動脈炎　［臨床試験数：22，薬物数：46（DrugBank：20），標的遺伝子数：22，標的パスウェイ数：111］

Searched query = "Takayasu arteritis", "Aortitis syndrome", "Pulseless disease"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 22 trial found

41. 巨細胞性動脈炎　［臨床試験数：108，薬物数：111（DrugBank：32），標的遺伝子数：33，標的パスウェイ数：121］

Searched query = "Giant cell arteritis", "Temporal arteritis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 108 trial found

42. 結節性多発動脈炎　［臨床試験数：14，薬物数：27（DrugBank：16），標的遺伝子数：26，標的パスウェイ数：102］

Searched query = "Polyarteritis nodosa", "PAN"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 14 trial found

45. 好酸球性多発血管炎性肉芽腫症　［臨床試験数：27，薬物数：41（DrugBank：18），標的遺伝子数：18，標的パスウェイ数：98］

Searched query = "Eosinophilic granulomatosis with Polyangiitis", "EGPA", "Eosinophilic multiple vasculitis granulomatous disease", "Allergic granulomatous angiitis", "Churg-Strauss syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 27 trial found

46. 悪性関節リウマチ　［臨床試験数：4,183，薬物数：2,538（DrugBank：401），標的遺伝子数：183，標的パスウェイ数：219］

Searched query = "Malignant rheumatoid arthritis", "Rheumatoid arthritis", "Rheumatoid arthritis with vasculitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 4,183 trial found

49. 全身性エリテマトーデス　［臨床試験数：827，薬物数：638（DrugBank：168），標的遺伝子数：108，標的パスウェイ数：191］

Searched query = "Systemic lupus erythematosus", "SLE"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 827 trial found

50. 皮膚筋炎／多発性筋炎　［臨床試験数：157，薬物数：207（DrugBank：76），標的遺伝子数：47，標的パスウェイ数：142］

Searched query = "Dermatomyositis", "Polymyositis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 157 trial found

53. シェーグレン症候群　［臨床試験数：234，薬物数：270（DrugBank：85），標的遺伝子数：49，標的パスウェイ数：174］

Searched query = "Sjogren syndrome", "Sjögren syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 234 trial found

55. 再発性多発軟骨炎　［臨床試験数：7，薬物数：10（DrugBank：9），標的遺伝子数：12，標的パスウェイ数：98］

Searched query = "Relapsing polychondritis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 7 trial found

56. ベーチェット病　［臨床試験数：68，薬物数：87（DrugBank：31），標的遺伝子数：36，標的パスウェイ数：113］

Searched query = "Behcet disease", "Behçet disease"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 68 trial found

58. 肥大型心筋症　［臨床試験数：92，薬物数：126（DrugBank：35），標的遺伝子数：40，標的パスウェイ数：141］

Searched query = "Hypertrophic cardiomyopathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 92 trial found

60. 再生不良性貧血　［臨床試験数：218，薬物数：362（DrugBank：81），標的遺伝子数：39，標的パスウェイ数：155］

Searched query = "Aplastic anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 218 trial found

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT00944749 (ClinicalTrials.gov)	July 2009	22/7/2009	Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment	Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment	Anemia, Aplastic;Anemia, Hypoplastic	Drug: h-ATG (ATGAM );Drug: Cyclosporine (Gengraf )	National Heart, Lung, and Blood Institute (NHLBI)	NULL	Completed	2 Years	82 Years	All	23	Phase 2	United States

61. 自己免疫性溶血性貧血　［臨床試験数：90，薬物数：72（DrugBank：23），標的遺伝子数：19，標的パスウェイ数：147］

Searched query = "Autoimmune hemolytic anemia", "AIHA", "WAIHA", "Cold agglutinin disease", "CAD", "Paroxysmal cold hemoglobinuria", "MAIHA", "Evans syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 90 trial found

62. 発作性夜間ヘモグロビン尿症　［臨床試験数：202，薬物数：120（DrugBank：25），標的遺伝子数：15，標的パスウェイ数：90］

Searched query = "Paroxysmal nocturnal hemoglobinuria", "PNH"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 202 trial found

63. 特発性血小板減少性紫斑病　［臨床試験数：311，薬物数：185（DrugBank：39），標的遺伝子数：43，標的パスウェイ数：132］

Searched query = "Idiopathic thrombocytopenic purpura", "Primary immune thrombocytopenia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 311 trial found

64. 血栓性血小板減少性紫斑病　［臨床試験数：74，薬物数：76（DrugBank：19），標的遺伝子数：15，標的パスウェイ数：57］

Searched query = "Thrombotic thrombocytopenic purpura", "TTP", "Upshaw-Schulman syndrome", "USS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 74 trial found

65. 原発性免疫不全症候群　［臨床試験数：413，薬物数：581（DrugBank：97），標的遺伝子数：68，標的パスウェイ数：202］

Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 413 trial found

90. 網膜色素変性症　［臨床試験数：103，薬物数：158（DrugBank：42），標的遺伝子数：52，標的パスウェイ数：107］

Searched query = "Retinitis pigmentosa", "Rod dystrophy", "Rod-Cone Dystrophy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 103 trial found

93. 原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］　［臨床試験数：230，薬物数：215（DrugBank：51），標的遺伝子数：34，標的パスウェイ数：107］

Searched query = "Primary biliary cholangitis", "Primary biliary cirrhosis", "PBC"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 230 trial found

95. 自己免疫性肝炎　［臨床試験数：44，薬物数：57（DrugBank：26），標的遺伝子数：18，標的パスウェイ数：108］

Searched query = "Autoimmune hepatitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 44 trial found

97. 潰瘍性大腸炎　［臨床試験数：2,269，薬物数：1,331（DrugBank：241），標的遺伝子数：114，標的パスウェイ数：181］

Searched query = "Ulcerative colitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 2,269 trial found

113. 筋ジストロフィー　［臨床試験数：567，薬物数：442（DrugBank：93），標的遺伝子数：55，標的パスウェイ数：151］

Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 567 trial found

162. 類天疱瘡（後天性表皮水疱症を含む。）　［臨床試験数：70，薬物数：117（DrugBank：46），標的遺伝子数：30，標的パスウェイ数：128］

Searched query = "Pemphigoid", "Epidermolysis bullosa acquisita"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 70 trial found

164. 眼皮膚白皮症　［臨床試験数：15，薬物数：57（DrugBank：34），標的遺伝子数：34，標的パスウェイ数：136］

Searched query = "Oculocutaneous albinism", "Hermansky-Pudlak syndrome", "Chediak-Higashi syndrome", "Griscelli syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 15 trial found

222. 一次性ネフローゼ症候群　［臨床試験数：234，薬物数：241（DrugBank：78），標的遺伝子数：59，標的パスウェイ数：185］

Searched query = "Primary nephrotic syndrome", "Minimal change nephrotic syndrome", "MCNS", "Membranous nephropathy", "Focal segmental glomerulosclerosis", "FSGS", "Membranoproliferative glomerulonephritis", "MPGN"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 234 trial found

226. 間質性膀胱炎（ハンナ型）　［臨床試験数：133，薬物数：151（DrugBank：46），標的遺伝子数：48，標的パスウェイ数：137］

Searched query = "Interstitial cystitis with Hunners ulcer", "Interstitial cystitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 133 trial found

228. 閉塞性細気管支炎　［臨床試験数：92，薬物数：125（DrugBank：34），標的遺伝子数：33，標的パスウェイ数：152］

Searched query = "Bronchiolitis obliterans", "Obliterating bronchiolitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 92 trial found

234. ペルオキシソーム病（副腎白質ジストロフィーを除く。）　［臨床試験数：38，薬物数：37（DrugBank：12），標的遺伝子数：12，標的パスウェイ数：42］

Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "PEX gene disorder", "Zellweger syndrome", "Neonatal adrenoleukodystrophy", "Rhizomelic chondrodysplasia punctata type 1", "RCDP type 1", "RCDP1", "Peroxisomal beta-oxidation enzyme deficiency", "Acyl-CoA oxidase deficiency", "AOX deficiency", "D-Bifunctional protein deficiency", "DBP deficiency", "Sterol carrier protein X deficiency", "SCPx deficiency", "2-methylacyl-CoA racemase deficiency", "Alpha-methylacyl-CoA racemase deficiency", "AMACR deficiency", "Refsum disease", "Plasmalogen biosynthesis enzyme deficiency", "Rhizomelic chondrodysplasia punctata type 2", "RCDP type 2", "RCDP2", "Rhizomelic chondrodysplasia punctata type 3", "RCDP type 3", "RCDP3", "Primary hyperoxaluria type 1", "PH1", "Acatalasemia", "Acatalasia", "Contiguous ABCD1/DXS1357E deletion syndrome", "CADDS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 38 trial found

269. 化膿性無菌性関節炎・壊疽性膿皮症・アクネ症候群　［臨床試験数：20，薬物数：26（DrugBank：10），標的遺伝子数：12，標的パスウェイ数：90］

Searched query = "Pyogenic arthritis", "Pyoderma gangrenosum", "Acne syndrome", "PAPA syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 20 trial found

274. 骨形成不全症　［臨床試験数：78，薬物数：87（DrugBank：19），標的遺伝子数：14，標的パスウェイ数：74］

Searched query = "Osteogenesis Imperfecta"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 78 trial found

283. 後天性赤芽球癆　［臨床試験数：17，薬物数：36（DrugBank：19），標的遺伝子数：16，標的パスウェイ数：91］

Searched query = "Acquired pure red cell aplasia", "Pure red cell aplasia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 17 trial found

284. ダイアモンド・ブラックファン貧血　［臨床試験数：37，薬物数：110（DrugBank：34），標的遺伝子数：22，標的パスウェイ数：121］

Searched query = "Diamond-Blackfan anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 37 trial found

285. ファンコニ貧血　［臨床試験数：56，薬物数：111（DrugBank：31），標的遺伝子数：30，標的パスウェイ数：151］

Searched query = "Fanconi anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 56 trial found

302. レーベル遺伝性視神経症　［臨床試験数：20，薬物数：14（DrugBank：4），標的遺伝子数：5，標的パスウェイ数：32］

Searched query = "Leber hereditary optic neuropathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 20 trial found

326. 大理石骨病　［臨床試験数：20，薬物数：52（DrugBank：14），標的遺伝子数：16，標的パスウェイ数：78］

Searched query = "Osteopetrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 20 trial found