Nemaline Myopathy
Conditions
Brief summary
The purpose of the study is to investigate if treatment with L-Tyrosine improves selected outcome measures of TNNT1 myopathy.
Interventions
DRUGL-Tyrosine
DRUGPlacebo
Sponsors
Hadassah Medical Organization
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
No minimum to 20 Years
Inclusion criteria
* Male or female patients * any age with TNNT1-related myopathy
Exclusion criteria
* Patients who are non-cooperative or parents/ legal guardians who are unwilling to sign consent form
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. goal attainment score | 3 month |
Countries
Israel
Outcome results
None listed