DDrare: Database of Drug Development for Rare Diseases

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

3 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
3	脊髄性筋萎縮症	1
11	重症筋無力症	0
12	先天性筋無力症候群	0

3. 脊髄性筋萎縮症　［臨床試験数：179，薬物数：102（DrugBank：26），標的遺伝子数：52，標的パスウェイ数：78］

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. 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

12. 先天性筋無力症候群　［臨床試験数：5，薬物数：7（DrugBank：3），標的遺伝子数：5，標的パスウェイ数：12］

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