Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. BridgeBio said Monday that an experimental ...

Fuchs’ dystrophy is an eye condition that causes the death of cells in the inner lining of the cornea. This may lead to the accumulation of fluid in the cornea and negatively affect vision. One of the ...

Nine people around the world who have been living with unexplained, muscular dystrophy-like symptoms may now have an answer for what is causing the progressive muscle weakness in their body's ...

More than 30 rare muscular dystrophy types cause progressive muscle weakness from inherited gene mutations. Combined, they affect about 1 in 5,000 to 8,000 people. Common subtypes vary by age of onset ...

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 ...

Duchenne muscular dystrophy (DMD) is the most common and one of the most severe types of muscular dystrophy. Symptoms usually start between ages 2 and 6, and muscle weakness worsens quickly. DMD ...

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 ...

The Food and Drug Administration approved a second drug for a debilitating form of muscular dystrophy, a surprise decision after the medication was rejected for safety concerns just four months ago.

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 ...