Brain Diseases, Metabolic - 50 Studies Found
Recruiting |
: Natural History Study of Cystathionine Beta-synthase Deficiency Homocystinuria (CBSDH) : Homocystinuria Due to CBS Deficiency : 2016-12-02 |
Active, not recruiting |
: MD1003-AMN MD1003 in Adrenomyeloneuropathy :
: 2016-11-09 :
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Recruiting |
: Newborn Screening for Adrenoleukodystrophy : Adrenoleukodystrophy : 2016-10-31 : Procedure: newborns testing for ALD Routine newborn screening dried blood spots sample is used to test i |
Not yet recruiting |
: Early Diagnosis Of Childhood Cerebral ALD : Adrenoleukodystrophy : 2016-10-25 |
Recruiting |
: GMP Drink for PKU Study : Phenylketonuria : 2016-08-08 : Dietary Supplement: Glycomacropeptide-based protein substitute |
Completed |
: Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin : Hereditary Tyrosinemia, Type I : 2016-04-20 :
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Completed |
: Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1) : Hereditary Tyrosinemia, Type I : 2016-04-21 :
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Completed |
: Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1) : Hereditary Tyrosinemia, Type I : 2016-04-21 : Drug: Nitisinone A single oral dose of Nitisinone 10 mg Tablet will be administered. |
Enrolling by invitation |
: Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product :
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