DDrare: Database of Drug Development for Rare Diseases

Amifampridine phosphate 10 mg oral tablet (DrugBank: Amifampridine, Phosphate)

3 diseases

ID	Disease name (Link within this page)	Number of trials
3	Spinal muscular atrophy	1
11	Myasthenia	0
12	Congenital myasthenic syndrome	0

3. Spinal muscular atrophy [ 179 clinical trials, 102 drugs, (DrugBank: 26 drugs), 52 drug target genes, 78 drug target pathways]

Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
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 / 179 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	NCT03819660 (ClinicalTrials.gov)	March 7, 2019	23/1/2019	Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3	Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3	Muscle Atrophy	Drug: Amifampridine Phosphate 10 MG Oral Tablet	Catalyst Pharmaceuticals, Inc.	NULL	Enrolling by invitation	6 Years	50 Years	All	12	Phase 2	Italy

11. Myasthenia [ 226 clinical trials, 172 drugs, (DrugBank: 45 drugs), 43 drug target genes, 118 drug target pathways]

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

12. Congenital myasthenic syndrome [ 5 clinical trials, 7 drugs, (DrugBank: 3 drugs), 5 drug target genes, 12 drug target pathways]

Searched query = "Congenital myasthenic syndrome", "End-plate acetylcholine receptor deficiency", "Sodium channel myasthenia", "End-plate acetylcholine esterase deficiency", "Dok-7 myasthenia"
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 / 5 trial found