Fuchs’ dystrophy is a type of eye disease that affects the cornea. Your cornea is the dome-shaped outer layer of your eye that helps you see. Fuchs’ dystrophy can cause your vision to decrease over ...

The US Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...

The full-length dystrophin gene (Fig. 2Aa) is predominantly expressed in skeletal and cardiac muscle with smaller amounts expressed in the brain. Sixty-five per cent of the mutations are deletions of ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Muscular dystrophy affects approximately 1 in 3,500 male births. Though rare in females, there are documented cases. It is usually diagnosed between 3 and 6 years of age and is degenerative, causing ...

Muscular dystrophy is a group of disorders that involve a progressive loss of muscle mass and consequent loss of strength. In general, the condition is caused by genetic variations that interfere with ...

Dystrophy is any condition in which a part of the body weakens or wastes away. In muscular dystrophy, the weakness is in the muscles. An inherited genetic mistake prevents the body from making a ...

Muscular dystrophy isn’t one disorder, but rather a group of more than 30 types of genetic conditions that cause progressive muscle weakness and loss. Although the signs and symptoms of muscular ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of a novel gene therapy for ...