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Clinical Trials for Adrenoleukodystrophy

• A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
• Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
• Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
• Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy
• A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN
• A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)
• Plasma Exchange With Albumin in AMN Patients
• Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy
• Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
• Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
• A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.
• Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
• MD1003-AMN MD1003 in Adrenomyeloneuropathy
• Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)
• Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy
• MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
• A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Childhood Cerebral Adrenoleukodystrophy (CCALD)
• Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)
• Human Placental-Derived Stem Cell Transplantation
• A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants
• The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)
• Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
• A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy
• HSCT for High Risk Inherited Inborn Errors
• Stem Cell Transplant for Inborn Errors of Metabolism
• Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
• Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
• Study of Bile Acids in Patients With Peroxisomal Disorders
• Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy