Adrenoleukodystrophy News and Research RSS Feed - Adrenoleukodystrophy News and Research

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.
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

A drug used to control Type II diabetes can help repair the spinal cords of mice suffering from the inherited disease adrenoleukodystrophy which, untreated, leads eventually to a paralysis, a vegetative state and death. [More]
IDIBELL signs patent licensing agreement with Minoryx

IDIBELL signs patent licensing agreement with Minoryx

The Bellvittge Biomedical Research Institute has signed a licensing agreement with the Spanish biotechnology company Minoryx of a patent for the treatment of X-linked adrenoleukodystrophy, a rare serious neurodegenerative disease which has no effective treatment. [More]
bluebird bio completes $60 million Series D financing

bluebird bio completes $60 million Series D financing

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, today announced the successful completion of a $60 million Series D financing. In this round, new investors Deerfield Partners, RA Capital, Ramius Capital Group, and two undisclosed blue chip public investment funds joined existing investors ARCH Venture Partners, Third Rock Ventures, TVM Capital, and Forbion Capital Partners. In addition, Shire plc joined the round as a strategic investor. [More]
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

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD). [More]
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

genOway and bluebird bio report today genOway acquisition of an exclusive worldwide license for the RMCE technology (developed at the Massachusetts Institute of Technology) in the field of genetically modified rodents. [More]
bluebird bio announces receipt of additional $30 million in financing

bluebird bio announces receipt of additional $30 million in financing

bluebird bio, an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced that the company has secured an additional $30 million in financing. [More]
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

A pediatric immunologist at The Children's Hospital of Philadelphia collaborated with European gene therapy researchers who achieved marked clinical improvements in two young children with Wiskott-Aldrich syndrome, a very rare but often severe immunodeficiency disorder. [More]
bluebird bio publishes LentiGlobin gene therapy clinical trial data results

bluebird bio publishes LentiGlobin gene therapy clinical trial data results

bluebird bio an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced publication in the journal Nature of its promising Phase 1/2 data highlighting positive results of LentiGlobin gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases. [More]
Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery technologies, announced today that it has received a National Institutes of Health (NIH) small business technology transfer (STTR) grant for a program on "Lentiviral Gene Therapy for Mucopolysaccharidosis." [More]

Genetix Pharmaceuticals announces completion of $35M Series B financing

Genetix Pharmaceuticals, a leading gene therapy company developing breakthrough treatments for severe genetic disorders, announced today that it has completed a $35 million Series B financing with new investors Third Rock Ventures and Genzyme Ventures joining TVM Capital, Forbion and Easton Capital. Proceeds from the financing will be used to advance current clinical programs, strengthen platform capabilities and further expand the team. [More]

Genetix Pharmaceuticals develops a gene therapy product to treat ALD

Genetix Pharmaceuticals, a leader in gene therapy of somatic stem cells, announced today the company’s clinical program to treat Adrenoleukodystrophy (ALD). The company is developing a gene therapy product in collaboration with Professor Patrick Aubourg and Doctor Nathalie Cartier of the National Institute of Health and Medical Research (INSERM). [More]
Gene therapy saves two children with adrenoleukodystrophy

Gene therapy saves two children with adrenoleukodystrophy

The ELA association and Zinedine Zidane, its emblematic ambassador, are proud to announce a world premiere: the results regaring the gene therapy in adrenoleukodystrophy conducted in France have just been published in the prestigious journal Science. Two children have been treated and their diseases have been halted. The children are doing well, which is unexpected for a disease destroying the brain in a few months. This discovery opens up treatment perspectives for numerous widespread diseases. [More]
Human stem cells show promise against fatal children's diseases

Human stem cells show promise against fatal children's diseases

Scientists have used human stem cells to dramatically improve the condition of mice with a neurological condition similar to a set of diseases in children that are invariably fatal, according to an article in the June issue of the journal Cell Stem Cell. [More]
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

New approaches to genetic disease, based on cells' own ability to correct themselves, will be outlined today at the annual conference of the European Society of Human Genetics in Amsterdam, The Netherlands. [More]
Study proves Lorenzo's Oil  prevents rare disease in boys

Study proves Lorenzo's Oil prevents rare disease in boys

U.S. researchers have confirmed that a treatment called Lorenzo's Oil can prevent the onset of a rare but devastating neurological disease in young boys, X-linked adrenoleukodystrophy or X-ALD. [More]
Lorenzo's oil prevents onset of pediatric neurological disorder

Lorenzo's oil prevents onset of pediatric neurological disorder

Use of Lorenzo's Oil in young boys who have been diagnosed with but are not yet showing signs of a pediatric neurological disorder known as X-linked adrenoleukodystrophy (X-ALD) may prevent the disease from developing in the body. According to a study of male children published today in the Archives of Neurology, use of the oil prevented onset of the disease in a majority of cases. [More]
Infants with Krabbe disease saved by cord blood stem cells

Infants with Krabbe disease saved by cord blood stem cells

Children with a fatal genetic disorder called Krabbe Disease can be saved and their brain development preserved if they receive stem cells from umbilical cord blood before symptoms of the disease develop, according to a study published in the current issue of the New England Journal of Medicine. [More]