Download 500 326 1 in 3500-5000

Drisapersen Clinical Trial Program
Duchenne Muscular Dystrophy
Changes in the dystrophin gene (mutations) that lead to the near
absence of dystrophin protein result in the most severe form of
The largest and longest Duchenne
clinical trial program submitted to the FDA.
dystrophin deficient muscular dystrophy, Duchenne muscular
dystrophy, also known as just Duchenne.
Since 2007
It is estimated to be present in
1 in 3,500-5,000
newborn boys with symptoms appearing as
early as the age of two.
Dystrophin protein plays an important structural role in the
performance of muscles. Without dystrophin, boys living with
Duchenne experience progressive muscle weakness, causing
serious medical complications including serious heart or
326
boys have
participated in
clinical trials with
drisapersen
9clinical studies, 70sites
3randomized 25countries
In
More than
in
including
placebo-controlled studies
respiratory-related complications, resulting in death in
early adulthood.
Drisapersen & Exon 51 Skipping
Drisapersen is an antisense oligonucleotide that induces exon
skipping to provide a molecular patch for dystrophin transcripts
produced by certain mutated dystrophin genes.
Exons are the parts of a gene that contain the instructions for
generating a protein. In applicable cases, skipping an exon
near the mutation allows for the production of a truncated but
functional dystrophin protein.
Representing
nearly
500
total patient
years on drisapersen
Drisapersen was granted
Breakthrough
Therapy designation by
the United States Food
and Drug Administration
(FDA) in June 2013
And granted
Orphan status
in the U.S., E.U.,
Australia and Japan