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Latest Adrenoleukodystrophy Stories

2014-04-29 08:36:49

GULF SHORES, Ala., April 29, 2014 /PRNewswire/ -- If successful, Neuralgene's (http://www.neuralgene.com) PRCN-323 treatment could significantly change the prognosis for everyone with the rare genetic disorder known as adrenoleukodystrophy, or ALD. The biotech company has recently celebrated a series of early successes in the treatment of other neurological conditions like ALS (Lou Gehrig's disease). Founder and CEO Jason Williams, M.D., believes his team is on the path toward a cure...

2010-05-18 10:45:00

GAITHERSBURG, Md., May 18 /PRNewswire/ -- 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." In this program, Lentigen will collaborate with Dr. R. Scott McIvor Professor, Department of Genetics, Cell Biology and...

2010-01-07 17:56:44

University of Alberta medical researchers have made a major breakthrough in understanding a group of deadly disorders that includes the disease made famous in the movie Lorenzo's Oil. Because this group of diseases is inherited, the discovery could help in screening carriers and lead to prevention or an effective treatment. Richard Rachubinski, in the Faculty of Medicine & Dentistry, is an expert on structures in cells called peroxisomes which are involved in breaking down fatty acids....

2009-11-06 10:01:09

The ELA Association represents the largest funder for research on this fatal brain disease The ELA association and Zinedine Zidane, its emblematic ambassador, are proud to announce a world premiere: the results regarding 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...

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2009-11-06 07:18:17

These findings appear in the November 6, 2009 issue of the journal Science, which is published by AAAS, the nonprofit science society. In a pilot study of two patients monitored for two years, an international team of researchers slowed the onset of the debilitating brain disease X-linked adrenoleukodystrophy (ALD) using a lentiviral vector to introduce a therapeutic gene into patient's blood cells. Although studies with larger cohorts of patients are needed, these results suggest that...

2009-11-06 07:03:59

Affected families achieve goal of saving children. Exciting results reported in Science The Stop ALD Foundation today applauded the investigators who are reporting in the current issue of Science successful results from the pioneering use of gene therapy for adrenoleukodystrophy (ALD), a potentially crippling and fatal brain disorder in young boys. "As an organization founded by families affected by ALD, we know too well the ravages that this disorder inflicts on its victims and the...

2008-10-31 12:00:12

Aldagen, a biopharmaceutical company, has received orphan drug designation for ALD-101 from the FDA. The designation was granted for the use of ALD-101 to improve patient outcomes by decreasing time to platelet and neutrophil engraftment in patients with inherited metabolic disorders undergoing umbilical cord blood transplantation. Aldagen is currently conducting a Phase III trial infusing ALD-101 in patients with inherited metabolic disorders undergoing umbilical cord blood...

2008-10-30 09:00:41

Aldagen, Inc. today announced that it has received orphan drug designation for ALD-101 from the U.S. Food and Drug Administration (FDA). The designation was granted for the use of ALD-101 to improve patient outcomes by decreasing time to platelet and neutrophil engraftment in patients with inherited metabolic disorders undergoing umbilical cord blood transplantation. Aldagen is currently conducting a Phase 3 trial infusing ALD-101 in patients with inherited metabolic disorders undergoing...

2005-07-11 22:35:00

BALTIMORE, Md. (July 11, 2005) "“ 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. For the first time, the study demonstrated the preventive effects of...


Word of the Day
grass-comber
  • A landsman who is making his first voyage at sea; a novice who enters naval service from rural life.
According to the OED, a grass-comber is also 'a sailor's term for one who has been a farm-labourer.'