Adrenoleukodystrophy
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2025-10-23Clinical Trial Update: The Myelin Disorders Biorepository Project
This clinical trials update describes the Myelin Disorders Biorepository Project (NCT03047369) from Children’s Hospital of Philadelphia, cur…
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CLINICAL_TRIALS research · 2025-09-08Clinical Trial Update: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells [Phase 1]
Phase 1 trial evaluating intrathecal umbilical cord blood-derived oligodendrocyte-like cells for several inherited metabolic brain diseases;…
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CLINICAL_TRIALS research · 2025-05-08Clinical Trial Update: Adrenoleukodystrophy National Registry Study
A clinical trial registry update indicates a National Registry Study for Adrenoleukodystrophy is currently recruiting.
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CLINICAL_TRIALS research · 2025-04-04Clinical Trial Update: Early Check: Expanded Screening in Newborns
An update on an ongoing clinical trial (NCT03655223) evaluating expanded newborn screening under the Early Check program, with enrollment by…
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CLINICAL_TRIALS trial_result · 2025-03-18Clinical Trial Update: A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients. [Phase 2, Phase 3]
The clinical trial update indicates completion of Phase 2 and Phase 3 studies evaluating MIN-102 for efficacy and safety in male patients wi…
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CLINICAL_TRIALS trial_result · 2025-02-20Clinical Trial Update: Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
This clinical trial update provides long-term follow-up data for participants with cerebral adrenoleukodystrophy who were treated with the L…
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CLINICAL_TRIALS withdrawal · 2024-12-16Clinical Trial Update: Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) [Phase 2]
Phase 2 trial of PXL065 for adrenomyeloneuropathy (AMN), a form of X-linked adrenoleukodystrophy, has been withdrawn.
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EMA withdrawal · 2024-11-20EMA Refused: Nezglyal (leriglitazone hydrochloride) — Minoryx Therapeutics S.L.
The EMA refused to grant marketing authorization for Nezglyal (leriglitazone hydrochloride) for the treatment of adrenoleukodystrophy, under…
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CLINICAL_TRIALS trial_result · 2023-09-15Clinical Trial Update: Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating conditioning regimens for allogeneic hematopoietic stem-cell transplantation has completed recruitment…
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CLINICAL_TRIALS trial_result · 2022-09-22Clinical Trial Update: Plasma Exchange With Albumin in AMN Patients [Phase 2, Phase 3]
A Phase 2/Phase 3 trial evaluating plasma exchange with albumin in patients with Adrenomyeloneuropathy and Adrenoleukodystrophy has complete…
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CLINICAL_TRIALS trial_result · 2022-05-31Clinical Trial Update: Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy [Phase 1, Phase 2]
Clinical trial update for autologous hematopoietic stem cell gene therapy targeting Metachromatic Leukodystrophy and Adrenoleukodystrophy in…
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EMA withdrawal · 2022-04-04EMA Withdrawn: Skysona (elivaldogene autotemcel) — bluebird bio (Netherlands) B.V.
EMA has withdrawn Skysona (elivaldogene autotemcel) from the market in the Netherlands for adrenoleukodystrophy.
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CLINICAL_TRIALS trial_result · 2020-10-23Clinical Trial Update: Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy [Phase 2]
Phase 2 trial investigating glycerol trierucate for adrenoleukodystrophy was terminated; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2019-07-11Clinical Trial Update: HSCT for High Risk Inherited Inborn Errors [Phase 2]
A Phase 2 study updating hematopoietic stem cell transplantation for high-risk inherited metabolic disorders shows completed status with res…
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CLINICAL_TRIALS trial_result · 2018-07-12Clinical Trial Update: Newborn Screening for Adrenoleukodystrophy
This clinical trial update reports on the completion of a newborn screening study for Adrenoleukodystrophy conducted by National Taiwan Univ…
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CLINICAL_TRIALS withdrawal · 2018-07-06Clinical Trial Update: Early Diagnosis Of Childhood Cerebral ALD
A clinical trial investigating early diagnosis of childhood cerebral adrenoleukodystrophy has been withdrawn from the registry. The record d…
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CLINICAL_TRIALS trial_result · 2017-10-09Clinical Trial Update: MD1003-AMN MD1003 in Adrenomyeloneuropathy [Phase 2, Phase 3]
Clinical trial MD1003 for Adrenomyeloneuropathy and Adrenoleukodystrophy has completed Phase 2/Phase 3.
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CLINICAL_TRIALS trial_result · 2015-03-25Clinical Trial Update: Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy [NA]
This update reports a completed randomized trial evaluating the combination of beta interferon and thalidomide in patients with adrenoleukod…
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CLINICAL_TRIALS trial_result · 2015-03-25Clinical Trial Update: Study of Bile Acids in Patients With Peroxisomal Disorders [NA]
A clinical trial evaluating bile acids for peroxisomal disorders has been terminated, involving diseases such as Infantile Refsum's Disease …
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CLINICAL_TRIALS trial_result · 2012-03-02Clinical Trial Update: A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy [Phase 3]
Phase III trial of Lorenzo's Oil for adrenomyeloneuropathy and adrenoleukodystrophy has been terminated.
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CLINICAL_TRIALS trial_result · 2011-06-10Clinical Trial Update: Stem Cell Transplant for Inborn Errors of Metabolism [Phase 2, Phase 3]
Phase 2/3 clinical trial update on stem cell transplant for inborn errors of metabolism; trial completed with results posted in 2011.
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CLINICAL_TRIALS research · 2009-02-09Clinical Trial Update: Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children [NA]
This entry describes a clinical study and gene mutation analysis of adrenoleukodystrophy in Taiwanese children, sponsored by Far Eastern Mem…