Adrenoleukodystrophy News and Research

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Adrenoleukodystrophy (ALD) is one of a group of genetic disorders called the leukodystrophies that cause damage to the myelin sheath, an insulating membrane that surrounds nerve cells in the brain.
Scientific breakthrough provides new hope for people living with multiple sclerosis

Scientific breakthrough provides new hope for people living with multiple sclerosis

Bluebird bio collaborates with Gritstone Oncology to develop novel cancer cell therapies

Bluebird bio collaborates with Gritstone Oncology to develop novel cancer cell therapies

Coconut oil diet increases vitality, lifespan of fruit flies with peroxisomal disorder

Coconut oil diet increases vitality, lifespan of fruit flies with peroxisomal disorder

Moms of children with rare genetic illness push for wider newborn screening

Moms of children with rare genetic illness push for wider newborn screening

Gene therapy effectively stabilizes progression of cerebral adrenoleukodystrophy, trial reveals

Gene therapy effectively stabilizes progression of cerebral adrenoleukodystrophy, trial reveals

Viking submits IND application to FDA to conduct VK2809 Phase 2 study in patients with LDL-C and fatty liver disease

Viking submits IND application to FDA to conduct VK2809 Phase 2 study in patients with LDL-C and fatty liver disease

Viking begins VK5211 Phase 2 trial in patients recovering from hip fracture

Viking begins VK5211 Phase 2 trial in patients recovering from hip fracture

Viking successfully completes safety, tolerability and pharmacokinetic study of VK5211 in elderly subjects

Viking successfully completes safety, tolerability and pharmacokinetic study of VK5211 in elderly subjects

Drug used to control Type II diabetes can help repair spinal cords of mice suffering from inherited disease

Drug used to control Type II diabetes can help repair spinal cords of mice suffering from inherited disease

IDIBELL signs patent licensing agreement with Minoryx

IDIBELL signs patent licensing agreement with Minoryx

bluebird bio completes $60 million Series D financing

bluebird bio completes $60 million Series D financing

FDA, EMA grant orphan drug designation to bluebird bio's gene therapy product for adrenoleukodystrophy

FDA, EMA grant orphan drug designation to bluebird bio's gene therapy product for adrenoleukodystrophy

genOway acquires worldwide exclusive license for RMCE technology from bluebird bio for genetically modified rodents

genOway acquires worldwide exclusive license for RMCE technology from bluebird bio for genetically modified rodents

bluebird bio announces receipt of additional $30 million in financing

bluebird bio announces receipt of additional $30 million in financing

Children's Hospital pediatric immunologist collaborates with European gene therapy researchers to study WAS syndrome

Children's Hospital pediatric immunologist collaborates with European gene therapy researchers to study WAS syndrome

bluebird bio publishes LentiGlobin gene therapy clinical trial data results

bluebird bio publishes LentiGlobin gene therapy clinical trial data results

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Genetix Pharmaceuticals announces completion of $35M Series B financing

Genetix Pharmaceuticals announces completion of $35M Series B financing

Genetix Pharmaceuticals develops a gene therapy product to treat ALD

Genetix Pharmaceuticals develops a gene therapy product to treat ALD

Gene therapy saves two children with adrenoleukodystrophy

Gene therapy saves two children with adrenoleukodystrophy