DDrare: Database of Drug Development for Rare Diseases

Gentamicin infusions twice a week for six months (DrugBank: Gentamicin)

3 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
36	表皮水疱症	0
113	筋ジストロフィー	1
299	嚢胞性線維症	0

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

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 567 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	NCT00451074 (ClinicalTrials.gov)	March 2007	21/3/2007	Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons	A Six Month Randomized, Clinical Trial of Gentamicin in Duchenne Muscular Dystrophy Subjects With Stop Codon Mutations	Duchenne Muscular Dystrophy	Drug: Gentamicin infusions twice a week for six months	Nationwide Children's Hospital	National Institutes of Health (NIH);National Institute of Neurological Disorders and Stroke (NINDS)	Completed	5 Years	20 Years	Male	12	Phase 1	United States

299. 嚢胞性線維症　［臨床試験数：1,592，薬物数：1,539（DrugBank：255），標的遺伝子数：81，標的パスウェイ数：162］

Searched query = "Cystic fibrosis"
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 / 1,592 trial found