Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study)
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The purpose of the registry/repository is to understand the natural history of tyrosinemia in our region and to provide a mechanism to store data and specimens to support the conduct of future research about hereditary tyrosinemia among the Arabs.
Condition or disease
Hereditary Tyrosinemia, Type I
The purpose of this study is to create an electronic registry of phenotypic, laboratory information, treatment and outcomes options for tyrosinemia type I. The registry is longitudinal in nature including retrospective clinical data from birth to the most recent encounter with all data entered in chronological fashion. The goals of this registry are the better understanding of the natural history and treatment outcomes of these patients and to determine/evaluate biochemical and clinical parameters for monitoring and prognosis of tyrosinemia type I.
Create a registry for tyrosinemia type I. [ Time Frame: 5 Years ]
This outcome is a binary 'yes/no' outcome as to whether or not this study can successfully create a repository with the intent to store data and specimens to support the conduct of future research on tyrosinemia type I.
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Layout table for eligibility information
Ages Eligible for Study:
up to 18 Years (Child, Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Patients with tyrosinemia type I seen by one of the participating sites.
Biochemical or molecular diagnosis of Tyrosinemia Type I.
Examined/followed by one of the participating sites.
Parental/guardian permission (informed consent) for participation.
Diagnosis of tyrosinemia has been excluded.
Not examined/followed by one of the participating sites.
Unwilling to provide informed consent for participation.