DDrare: Database of Drug Development for Rare Diseases

111. 先天性ミオパチー
［臨床試験数：7，薬物数：13（DrugBank：4），標的遺伝子数：1，標的パスウェイ数：8］

Searched query = "Congenital myopathy", "Nemaline myopathy", "Central core disease", "Minicore myopathy", "Myotubular myopathy", "Centronuclear myopathy", "Congenital fiber-type disproportion myopathy"
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.

Drug = AT132; Acetylcysteine; DYN101; L-Tyrosine; N-acetylcysteine; RAAV8-Des-hMTM1; SALBUTAMOL SULFATE; Salbutamol; Sulfate; Tyrosine; Ventoline; Ventoline oral solution 0,4mg/ml; Ventoline tablet 2mg

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	NCT02035501 (ClinicalTrials.gov)	January 2014	2/7/2013	Treatment of TNNT1-Myopathy With L-Tyrosine.	Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial.	Nemaline Myopathy	Drug: L-Tyrosine;Drug: Placebo	Hadassah Medical Organization	NULL	Enrolling by invitation	N/A	20 Years	Both	10	Phase 2	Israel

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

NCT02035501
(ClinicalTrials.gov)

January 2014

2/7/2013

Treatment of TNNT1-Myopathy With L-Tyrosine.

Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial.

Nemaline Myopathy

Drug: L-Tyrosine;Drug: Placebo

Hadassah Medical Organization

NULL

Enrolling by invitation

N/A

20 Years

Both

Phase 2

Israel