DDrare: Database of Drug Development for Rare Diseases

Cyclophosphamide (drug) (DrugBank: Cyclophosphamide)

44 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
13	多発性硬化症／視神経脊髄炎	1
14	慢性炎症性脱髄性多発神経炎／多巣性運動ニューロパチー	0
16	クロウ・深瀬症候群	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
35	天疱瘡	0
36	表皮水疱症	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
43	顕微鏡的多発血管炎	0
44	多発血管炎性肉芽腫症	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
51	全身性強皮症	0
52	混合性結合組織病	0
56	ベーチェット病	0
60	再生不良性貧血	0
62	発作性夜間ヘモグロビン尿症	0
65	原発性免疫不全症候群	0
66	IgA腎症	0
84	サルコイドーシス	0
85	特発性間質性肺炎	0
93	原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］	0
96	クローン病	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
172	低ホスファターゼ症	0
222	一次性ネフローゼ症候群	0
224	紫斑病性腎炎	0
256	筋型糖原病	0
265	脂肪萎縮症	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
288	自己免疫性後天性凝固因子欠乏症［自己免疫性出血病XIII (~2017.3)］	0
326	大理石骨病	0
331	特発性多中心性キャッスルマン病	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

13. 多発性硬化症／視神経脊髄炎　［臨床試験数：3,050，薬物数：2,147（DrugBank：348），標的遺伝子数：244，標的パスウェイ数：228］

Searched query = "Multiple sclerosis/Neuromyelitis optica", "Multiple sclerosis", "Neuromyelitis optica", "MS", "NMOSD", "Devic disease", "Balo concentric sclerosis", "Baló concentric sclerosis"
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 / 3,050 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	NCT00241254 (ClinicalTrials.gov)	December 2005	17/10/2005	Efficacy of Cyclophosphamide Versus Methylprednisolone in Patients With Secondary Progressive Multiple Sclerosis	A Double-blind, Two-arm, Multicenter, Randomized Trial to Evaluate Efficacy of Cyclophosphamide Versus Methylprednisolone in Patients With Recent Secondary Progressive Multiple Sclerosis: P.R.OM.E.S.S Study	Multiple Sclerosis, Chronic Progressive	Drug: Cyclophosphamide (drug);Drug: Methylprednisolone (drug)	University Hospital, Bordeaux	Ministry of Health, France	Completed	18 Years	65 Years	Both	138	Phase 3	France

14. 慢性炎症性脱髄性多発神経炎／多巣性運動ニューロパチー　［臨床試験数：145，薬物数：139（DrugBank：23），標的遺伝子数：12，標的パスウェイ数：21］

Searched query = "Chronic inflammatory demyelinating polyneuropathy", "Chronic inflammatory demyelinating poly (radiculo) neuropathy", "Multifocal motor neuropathy", "CIDP"
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 / 145 trial found

16. クロウ・深瀬症候群　［臨床試験数：12，薬物数：18（DrugBank：8），標的遺伝子数：5，標的パスウェイ数：79］

Searched query = "Crow-Fukase syndrome", "POEMS syndrome", "Polyneuropathy, organomegaly, endocrinopathy, m-protein, and skin changes syndrome", "Takatsuki disease", "PEP syndrome", "Polyneuropathy, endocrinopathy, plasma cell dyscrasia 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 / 12 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

35. 天疱瘡　［臨床試験数：87，薬物数：114（DrugBank：37），標的遺伝子数：17，標的パスウェイ数：158］

Searched query = "Pemphigus"
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 / 87 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

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

43. 顕微鏡的多発血管炎　［臨床試験数：85，薬物数：80（DrugBank：19），標的遺伝子数：13，標的パスウェイ数：85］

Searched query = "Microscopic polyangiitis", "MPA"
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 / 85 trial found

44. 多発血管炎性肉芽腫症　［臨床試験数：84，薬物数：88（DrugBank：22），標的遺伝子数：13，標的パスウェイ数：37］

Searched query = "Wegener granulomatosis", "Multiple vasculitis granulomatous disease", "Granulomatosis with polyangiitis"
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 / 84 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

51. 全身性強皮症　［臨床試験数：466，薬物数：536（DrugBank：142），標的遺伝子数：110，標的パスウェイ数：210］

Searched query = "Scleroderma", "Systemic sclerosis", "SSc", "dcSSc", "lcSSc"
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 / 466 trial found

52. 混合性結合組織病　［臨床試験数：4，薬物数：6（DrugBank：3），標的遺伝子数：1，標的パスウェイ数：1］

Searched query = "Mixed connective tissue 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 / 4 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

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: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 218 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

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

66. IgA腎症　［臨床試験数：199，薬物数：214（DrugBank：57），標的遺伝子数：32，標的パスウェイ数：128］

Searched query = "IgA nephropathy", "IgA nephritis", "Berger disease", "IgA-IgG nephropathy"
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 / 199 trial found

84. サルコイドーシス　［臨床試験数：143，薬物数：221（DrugBank：79），標的遺伝子数：82，標的パスウェイ数：165］

Searched query = "Sarcoidosis"
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 / 143 trial found

85. 特発性間質性肺炎　［臨床試験数：514，薬物数：377（DrugBank：108），標的遺伝子数：97，標的パスウェイ数：204］

Searched query = "Idiopathic interstitial pneumonia", "IIPs", "Idiopathic pulmonary fibrosis", "IPF", "Usual interstitial pneumonia", "UIP", "Non-specific interstitial pneumonia", "NSIP", "Acute interstitial pneumonia", "AIP", "Diffuse alveolar damage", "DAD", "COP", "Organizing pneumonia", "OP", "Desquamative interstitial pneumonia", "DIP", "Respiratory bronchiolitis - associated interstitial lung disease", "RB-ILD", "Lymphocytic interstitial pneumonia", "LIP"
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 / 514 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

96. クローン病　［臨床試験数：2,209，薬物数：1,276（DrugBank：240），標的遺伝子数：166，標的パスウェイ数：210］

Searched query = "Crohn disease", "Terminal ileitis"
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 / 2,209 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

172. 低ホスファターゼ症　［臨床試験数：32，薬物数：18（DrugBank：4），標的遺伝子数：3，標的パスウェイ数：6］

Searched query = "Hypophosphatasia"
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 / 32 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

224. 紫斑病性腎炎　［臨床試験数：10，薬物数：41（DrugBank：19），標的遺伝子数：15，標的パスウェイ数：56］

Searched query = "Purpura nephritis"
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 / 10 trial found

256. 筋型糖原病　［臨床試験数：161，薬物数：114（DrugBank：23），標的遺伝子数：26，標的パスウェイ数：106］

Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
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 / 161 trial found

265. 脂肪萎縮症　［臨床試験数：109，薬物数：164（DrugBank：59），標的遺伝子数：26，標的パスウェイ数：94］

Searched query = "Lipodystrophy", "Berardinelli-Seip syndrome", "Lawrence syndrome", "Barraquer-Simons 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 / 109 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

288. 自己免疫性後天性凝固因子欠乏症［自己免疫性出血病XIII (~2017.3)］　［臨床試験数：189，薬物数：219（DrugBank：29），標的遺伝子数：18，標的パスウェイ数：26］

Searched query = "Autoimmune acquired coagulation factor deficiency", "Coagulation factor deficiency", "Factor XIII deficiency", "Factor VIII deficiency", "Acquired hemophilia A", "von Willebrand Disease", "Factor V deficiency"
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 / 189 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

331. 特発性多中心性キャッスルマン病　［臨床試験数：30，薬物数：44（DrugBank：23），標的遺伝子数：30，標的パスウェイ数：148］

Searched query = "Idiopathic multicentric castleman disease", "Castleman 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 / 30 trial found