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Brain Diseases, Metabolic, Inborn - 25 Studies Found
Estado | Estudiar |
Recruiting |
Nombre del estudio: Newborn Screening for Adrenoleukodystrophy Condición: Adrenoleukodystrophy Fecha: 2016-10-31 Intervenciones: Procedure: newborns testing for ALD Routine newborn screening dried blood spots sample is used to test i |
Not yet recruiting |
Nombre del estudio: Early Diagnosis Of Childhood Cerebral ALD Condición: Adrenoleukodystrophy Fecha: 2016-10-25 |
Recruiting |
Nombre del estudio: GMP Drink for PKU Study Condición: Phenylketonuria Fecha: 2016-08-08 Intervenciones: Dietary Supplement: Glycomacropeptide-based protein substitute |
Completed |
Nombre del estudio: Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin Condición: Hereditary Tyrosinemia, Type I Fecha: 2016-04-20 Intervenciones:
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Completed |
Nombre del estudio: Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1) Condición: Hereditary Tyrosinemia, Type I Fecha: 2016-04-21 Intervenciones:
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Completed |
Nombre del estudio: Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1) Condición: Hereditary Tyrosinemia, Type I Fecha: 2016-04-21 Intervenciones: Drug: Nitisinone A single oral dose of Nitisinone 10 mg Tablet will be administered. |
Enrolling by invitation |
Nombre del estudio: Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product Condición:
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Not yet recruiting |
Nombre del estudio: Urease Inhibitor Drug Treatment for Urea Cycle Disorders Condición:
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Not yet recruiting |
Nombre del estudio: Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency: Condición: Pyruvate Dehydrogenase Complex Deficiency Fecha: 2015-11-17 Intervenciones:
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