Lysogene, based in Paris, has entered into a strategic collaboration with UMass Medical School and Auburn University of Auburn, Alabama, to develop preclinical studies using an adeno-associated virus (AAV) on GM1-gangliosidosis, "a rare, inherited disorder
characterized by severe neurological impairment,'' according to a press release from Lysogene.
PRENATAL TESTING is available to pregnant women for common inherited disorders
and other genetic conditions for which their child may be at risk.
The therapy is meant for treating a very rare inherited disorder
called lipoprotein lipase (LPL) deficiency in patients suffering from severe pancreatitis attacks.
Downs Syndrome is by far the most common genetically inherited disorder
in this country.
Scientists have discovered that molecules called reactive oxygen species (ROS) produced by the energy factories, or mitochondria, in cells, may play a role in a rare inherited disorder
in which uncontrolled inflammation damages the body's tissues.
The implant is designed to help patients with retinitis pigmentosa, an inherited disorder
which gradually destroys the retina.
This inherited disorder
can be passed on if both parents are carriers for the altered gene after medics worked with them and educated them, the family were able to have carrier testing, make decisions about life partners as well as access early testing in pregnancy.
Wilson's disease as such is a rare autosomal recessive inherited disorder
of copper metabolism.
The study is the first to look at brain function in adults with sickle cell disease, and it may mean new drugs are needed to protect the brains of patients with the inherited disorder
That drug treats an inherited disorder
known as Fabry disease, which is caused by the buildup of a particular type of fat in the body's cells.
The test was added to the University Hygienic Laboratory at the recommendation of the state's Congenital and Inherited Disorder
Fuchs' dystrophy is an inherited disorder
of the eye's cornea--the clear "window" that allows light to enter the eye.
Myozyme is the only treatment for Pompe disease--a severe, progressively debilitating and life-threatening inherited disorder
affecting a very small number of people throughout the world.
PRIMORDIAL dwarfism is a very rare inherited disorder
and the cause is unknown.
Stewart Bradley, 50, had the inherited disorder
Pseudoxanthoma elasticum (PXE), which causes fragmentation and mineralisation of elastic fibres in some tissues.