Werner is CEO of Alltrna and CEO-partner at Flagship Pioneering. Duchenne muscular dystrophy has seen more progress in the past 15 months (give or take) than in the last couple of decades combined. In ...

Duchenne muscular dystrophy (DMD) is a genetic disease in which muscle cells fail to produce a protein called dystrophin. Unlike some other forms of muscular dystrophy, in Duchenne there is a complete ...

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 ...

A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an experimental compound ...

Muscular dystrophy is a group of more than 30 inherited conditions that cause progressive muscle weakness and loss. Some forms of muscular dystrophy are diagnosed in infancy or childhood, while the ...

Duchenne muscular dystrophy (DMD) symptoms often start by age 2, with delays in developmental milestones like walking well by 18 months. Common early DMD clues include Gower’s sign, toe walking, ...