Leukodystrophy News and Research

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Leukodystrophies are inherited disorders that are characterized by a progressive breakdown (demyelination or hypomyelination) of the white matter in the central nervous system, caused by different mechanisms involving myelin proteins, as well as lipid and organic acid metabolism. They have to be distinguished from the “umbrella” term leukoencephalopathy, which is used to delineate any disease of the white matter.

Researchers agree to test new stem cell gene therapy for Sanfilippo disease in human trial

Scientists in Manchester, who have developed a stem cell gene therapy to reverse a fatal childhood illness, have agreed to work with a new therapeutics company to test it in a human trial.

11 May 2016

New tests could improve diagnosis of two rare childhood diseases

For the first time, researchers have developed tests that could improve the diagnosis of two rare childhood diseases known as congenital disorders of glycosylation (CDGs) and metachromatic leukodystrophy, and that could even lead to new treatments for CDGs.

5 Jan 2016

Researchers develop new method to capture how brain cells interact

Interactions between brain cells hold the key to healthy brain function and cognition, but many of those interactions are notoriously difficult to study.

21 Sep 2015

International research team identifies new gene associated with 4H leukodystrophy

Leukodystrophies are deadly neurodegenerative diseases that affect one in 7,000 children and remain incurable. These genetic diseases attack myelin or the "insulating rubber sheath" surrounding neurons, which leads to deteriorating health for affected children.

9 Jul 2015

UC Davis researchers settle long-standing controversy surrounding Canavan disease

UC Davis investigators have settled a long-standing controversy surrounding the molecular basis of an inherited disorder that historically affected Ashkenazi Jews from Eastern Europe but now also arises in other populations of Semitic descent, particularly families from Saudi Arabia.

28 Apr 2015

FDA clears ArmaGen's AGT-182 IND application for treatment of Hunter syndrome

ArmaGen, Inc., a privately held biotechnology company focused on developing novel therapies to treat severe neurological disorders, announced today that the Investigational New Drug (IND) application for the company's lead product candidate, AGT-182 for the treatment of Hunter syndrome, has been accepted by the U.S. Food and Drug Administration and is now active.

11 Dec 2014

State highlights: Calif.'s Prop. 45 fight; new benefits for same sex couples in N.C.; Aetna sheds jobs after Medicaid drop

A selection of health policy stories from California, North Carolina, Delaware, New York, Georgia, Pennsylvania, New Jersey, Florida and Kansas.

17 Oct 2014

Cancer drug shows promise in treating blood cell disorders

Scientists working to make gene therapy a reality have solved a major hurdle: how to bypass a blood stem cell's natural defenses and efficiently insert disease-fighting genes into the cell's genome.

27 Jun 2014

Researchers develop adjuvant which enhances effect of virus transduction

Lentiviruses, which belong to the family of retroviruses, are used as vectors to exchange genetic material in cells and can be used to replace a defective gene as defined by gene therapy.

5 Mar 2014

Manchester scientists treat fatal genetic brain disease using stem cell gene therapy

Scientists from The University of Manchester have used stem cell gene therapy to treat a fatal genetic brain disease in mice for the first time.

From The University of Manchester 25 Jul 2013

'Defanged' HIV used to deliver gene therapy

Researchers are having success using a "defanged" version of HIV to deliver gene therapy to children suffering from genetic diseases, a development that could improve other such care.

15 Jul 2013

First Edition: July 12, 2013

Today's early morning highlights from the major news organizations, including articles on data tracking of doctors and comparing the rollout of the Medicare Part D drug plans to implementation of the federal health law.

12 Jul 2013

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.

10 Jun 2013

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.

8 May 2013

New treatment strategy for patients with CMT disease on the horizon

A potential new treatment strategy for patients with Charcot-Marie-Tooth disease is on the horizon, thanks to research by neuroscientists now at the University at Buffalo's Hunter James Kelly Research Institute and their colleagues in Italy and England.

29 Apr 2013

Reprogrammed human skin cells highly effective in treating myelin disorders

A study out today in the journal Cell Stem Cell shows that human brain cells created by reprogramming skin cells are highly effective in treating myelin disorders, a family of diseases that includes multiple sclerosis and rare childhood disorders called pediatric leukodystrophies.

10 Feb 2013

Parents must be considered during expansion of genetic screening programs for newborns

Parents must be considered when states decide to expand genetic screening programs for newborns, according to a new study that looked at mandatory testing panels and political pressure by advocacy groups.

22 Jun 2012

Study identifies function of GlialCAM gene in brain edema

A study of the pathophysiology of this rare disease has uncovered one mechanism that destabilizes the homeostatic balance of brain cells causing edema.

20 Mar 2012

Age-associated decline of remyelination process may be reversible

New research highlights the possibility of reversing ageing in the central nervous system for multiple sclerosis (MS) patients. The study is published today, 06 January, in the journal Cell Stem Cell.

7 Jan 2012

Polystyrene surface triples number of human ES and iPS cells in culture

By irradiating typical polystyrene lab plates with ultraviolet (UV) waves, Whitehead Institute and MIT scientists have created a surface capable of tripling the number of human embryonic stem (ES) and induced pluripotent stem (iPS) cells that can be grown in culture by current methods. Use of this novel surface also eliminates the need for layers of mouse "feeder cells" to support ES- and iPS-cell growth.

8 Nov 2011