The American authorities have granted permission for a group of nine children with Canavan disease
to be treated.
: Canavan disease
is a fatal neurological disorder that belongs to a group of genetic disorders called leukodystrophies, characterized by the abnormal development or degeneration of myelin.
is a progressive degenerative disorder of the brain and nervous system.
18, 2015 /PRNewswire-USNewswire/ -- Turing Pharmaceuticals AG, a privately-held biopharmaceutical company focused on developing and commercializing innovative treatments for serious diseases and conditions, today announced a Sponsored Research Agreement with the University of Toledo to discover and develop new treatments for Canavan Disease
Tay-Sachs disease, Canavan disease
, familial dysautonomia, and cystic fibrosis) as well as the 10 disorders recommended for routine screening by the American College of Medical Genetics (ACMG).
Gao, director of the school's Gene Therapy Center, which is developing a treatment for a fatal condition called Canavan disease
6) Various differential diagnoses that were considered included Canavan disease
, which is characterised by a combination of macroencephaly, extensive cerebral white matter changes (without frontal preponderance) and basal ganglia abnormalities.
Apparently normal at birth, babies with Canavan Disease
develop an enlarged head, mental retardation, feeding difficulties and seizures.
At 6 months, Jacob was diagnosed with Canavan disease
, a rare and fatal genetic brain disorder that interferes with the natural production of myelin, the white matter in the brain responsible for transmitting nerve impulses from one part of the body to another.
HADERA, Israel, January 27 /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