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Latest Leukodystrophies Stories

2014-07-27 23:01:10

LifeScienceIndustryResearch.com adds “Metachromatic Leukodystrophy (MLD) Global Clinical Trials Review, H2, 2014” to its store. This report provides elemental information and data relating to the clinical trials on MLD. Dallas, TX (PRWEB) July 27, 2014 Metachromatic leukodystrophy (MLD, also called Arylsulfatase A deficiency) is a lysosomal storage disease which is commonly listed in the family of leukodystrophies as well as among the sphingolipidoses as it affects the metabolism of...

Two Genetic Diseases Cured Through HIV
2013-07-12 05:01:10

[ Watch the Video: Gene Therapy Used By San Raffaele Telethon Institute For Gene Therapy ] redOrbit Staff & Wire Reports - Your Universe Online The virus that causes AIDS has been used to cure a pair of different genetic diseases, according to research published Thursday in the journal Science. In the studies, Luigi Naldini, director of the San Raffaele Telethon Institute for Gene Therapy (TIGET) in Italy, and his colleagues report they successfully used HIV to cure metachromatic...

2010-01-19 08:00:00

HADERA, Israel, Jan. 19 /PRNewswire/ -- Yulia and Gili Ben-Moshe, who live in a small town in Israel, were confronted with every parent's nightmare: their third child, Lavi then three months old, was diagnosed with Canavan Disease. (Photo: http://www.newscom.com/cgi-bin/prnh/20100119/HS38083 ) Children with Canavan Disease first lose their sense of sight, then their hearing and their motor functions. Usually, before age 4, the disease kills off their brain cells and the children die. It...

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...

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
toccata
  • In music, a work for a keyboard-instrument, like the pianoforte or organ, originally intended to utilize and display varieties of touch: but the term has been extended so as to include many irregular works, similar to the prelude, the fantasia, and the improvisation.
This word is Italian in origin, coming from the feminine past participle of 'toccare,' to touch.
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