What is Duchenne muscular dystrophy (DMD)?
Duchenne muscular dystrophy (DMD) is a rare genetic disorder which is more commonly observed in boys. It is a type of muscular dystrophy. Muscular dystrophy involves weakening of muscles and reduction in flexibility. Duchenne muscular dystrophy (DMD) is the most common type of muscular. . . .+
Duchene muscular dystrophy (DMD) is an inherited progressive form of muscular dystrophy. It causes muscle degeneration or atrophy (of skeletal and heart muscles) and results in weakness. Muscle weakness may begin at an age of 3 or 4 years in the pelvic area, hips or legs, and shoulders.
There are more than hundred different disorders similar to muscular dystrophy. Of them a few are considered a type of muscular dystrophy. The exact prognosis depends much on the type of disease that affects an individual. There is no cure for any form of muscular dystrophy, but treatment can help prevent or reduce problems.
How long a person with Duchenne muscular dystrophy lives depends on many factors. Kids with DMD usually did not survive beyond their teen years until recently. But, with advances in cardiac and respiratory care, life expectancy has increased and is further increasing with more research and clinical trials going on.
The primary objective of gene therapy is to cure a patient with DMD completely. There is some research going on that is aimed for delivering a functional, full-size dystrophin gene to muscle cells using a harmless virus. Until now, research has been limited to delivering smaller mini or micro dystrophin genes.