PatientCrossroads CMD


           

Use of Tyrosine in Nemaline Myopathy

Study start: January 2014

Purpose
The aim of this study is to gather information on the use of tyrosine in individuals with muscle problems. Tyrosine is commonly used in individuals with nemaline myopathy. Information about side effects and benefits of this medication is limited. We wish to gather information about tyrosine from parents of children with nemaline myopathy and from individuals with nemaline myopathy.
 
Participation
The study consent and questionnaire are available online. It is anticipated that it will take approximately 20 minutes to complete this survey. The survey and consent are also available as word documents which may be printed and completed manually, or completed electronically and returned via email. If you would prefer to complete one of these versions of the questionnaire and consent, please contact Dr Sarah Sandaradura, the study co-ordinator.
 
Eligibility:
1. Diagnosis of nemaline myopathy by muscle biopsy or genetic testing in affected child (age <18 years) or in affected individual (age >18 years).
2.  Consent must be provided by parent/caregiver (affected individual’s age less than 18 years or for those individuals greater than 18 years with learning disabilities or inability to physically access consent) or affected individual (age greater than 18 years)
 
To participate in this study please contact:
Dr Sarah Sandaradura, Institute for Neuroscience and Muscle Research, Children’s Hospital at Westmead, Sydney, Australia
Phone: 61 2 9845 0000
Email:  This e-mail address is being protected from spambots. You need JavaScript enabled to view it
 
For additional information on this study or to obtain an information sheet with additional information on use of tyrosine in neuromuscular conditions, please contact Dr Sarah Sandaradura via email, as above.
 
Participation is at no cost to the patient and there is no compensation for participation. This study has been approved by the Ethics and Governance committees of the Sydney Children’s Hospitals Network (Australia).