Disease Team Therapy Development - Research Duchenne muscular
dystrophy (DMD) is the most common muscular dystrophy and one of
the most common fatal genetic disorders. Approximately one in
every 3,500 boys worldwide is affected with DMD. Extrapolating
from population based studies, there are over 15,000 people
currently living with DMD in the US. DMD is a devastating and
incurable muscle-wasting disease caused by genetic mutations in
the gene that codes for dystrophin, a protein that plays a key
role in muscle cell health. Children are typically weaker than
normal by age three, and progressive muscle weakness of the
legs, pelvis, arms, neck and other areas result in most patients
requiring full-time use of a wheelchair by age 11. Eventually,
the disease progresses to complete paralysis and increasing
difficulty in breathing due to respiratory muscle dysfunction
and heart failure. The condition is terminal, and death usually
occurs before the age of 25. While corticosteroids can slow
disease progression and su