Peroxisomal Disorders - 25 Studies Found
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 |
Enrolling by invitation |
: Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product :
: 2015-08-12 : Genetic: transduced CD34+ hematopoietic stem cell Autologous hematopoietic stem cells (HSCs) collected f |
Recruiting |
: Inherited Retinal Degenerative Disease Registry :
: 2015-04-03 |
Withdrawn |
: MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD) : Cerebral Adrenoleukodystrophy : 2015-03-27 : Biological: Mesenchymal Stem Cells |
Recruiting |
: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells :
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Available |
: Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy : X-linked Adrenoleukodystrophy : 2014-05-05 : Drug: Lorenzo's Oil Oral use of Lorenzo's oil to lower very long chain fatty acids |