Adrenoleukodystrophy News and Research

RSS
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. People with ALD accumulate high levels of saturated, very long chain fatty acids (VLCFA) in the brain and adrenal cortex because they do not produce the enzyme that breaks down these fatty acids in the normal manner. The loss of myelin and the progressive dysfunction of the adrenal gland are the primary characteristics of ALD. ALD has two subtypes. The most common is the X-linked form (X-ALD), which involves an abnormal gene located on the X-chromosome.
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

Human stem cells show promise against fatal children's diseases

Human stem cells show promise against fatal children's diseases

New approaches to genetic disease, based on cells' own ability to correct themselves

New approaches to genetic disease, based on cells' own ability to correct themselves

Study proves Lorenzo's Oil  prevents rare disease in boys

Study proves Lorenzo's Oil prevents rare disease in boys

Lorenzo's oil prevents onset of pediatric neurological disorder

Lorenzo's oil prevents onset of pediatric neurological disorder

Infants with Krabbe disease saved by cord blood stem cells

Infants with Krabbe disease saved by cord blood stem cells